Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
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Title
Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
Authors
Keywords
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Journal
Biomolecules
Volume 11, Issue 4, Pages 611
Publisher
MDPI AG
Online
2021-04-21
DOI
10.3390/biom11040611
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Note: Only part of the references are listed.- Safety and efficacy of an rAd26 and rAd5 vector-based heterologous prime-boost COVID-19 vaccine: an interim analysis of a randomised controlled phase 3 trial in Russia
- (2021) Denis Y Logunov et al. LANCET
- A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
- (2020) Li Ou et al. GENE THERAPY
- AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer
- (2020) Helena Costa Verdera et al. MOLECULAR THERAPY
- Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses
- (2020) Jonathan M. Levy et al. Nature Biomedical Engineering
- A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I
- (2020) Li Ou et al. MOLECULAR THERAPY
- Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
- (2020) James M. Wilson et al. HUMAN GENE THERAPY
- Lysosomal storage diseases: current therapies and future alternatives
- (2020) Andrés Felipe Leal et al. JOURNAL OF MOLECULAR MEDICINE-JMM
- Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy
- (2020) Saori Miwa et al. MOLECULAR GENETICS AND METABOLISM
- Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
- (2020) Hannah G. Lahey et al. MOLECULAR THERAPY
- Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editing
- (2020) Samantha G. Scharenberg et al. Nature Communications
- Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration
- (2020) Christian Hinderer et al. Molecular Therapy-Methods & Clinical Development
- Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease
- (2020) Merel Stok et al. Molecular Therapy-Methods & Clinical Development
- Novel therapies for mucopolysaccharidosis type III
- (2020) Berna Seker Yilmaz et al. JOURNAL OF INHERITED METABOLIC DISEASE
- AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
- (2020) Tyler B. Johnson et al. MOLECULAR THERAPY
- Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
- (2020) Giuseppa Piras et al. Molecular Therapy-Methods & Clinical Development
- Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10
- (2020) Lalitha R. Belur et al. Molecular Genetics and Metabolism Reports
- Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector
- (2020) Maria Dahl et al. Molecular Therapy-Methods & Clinical Development
- Lysosomal storage disorders – challenges, concepts and avenues for therapy: beyond rare diseases
- (2019) André R. A. Marques et al. JOURNAL OF CELL SCIENCE
- Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease
- (2019) Jeong-A Lim et al. Molecular Therapy-Methods & Clinical Development
- CAV-2 Vector Development and Gene Transfer in the Central and Peripheral Nervous Systems
- (2019) Danila del Rio et al. Frontiers in Molecular Neuroscience
- Therapeutic landscape for Batten disease: current treatments and future prospects
- (2019) Tyler B. Johnson et al. Nature Reviews Neurology
- Macroglossia, Motor Neuron Pathology, and Airway Malacia Contribute to Respiratory Insufficiency in Pompe Disease: A Commentary on Molecular Pathways and Respiratory Involvement in Lysosomal Storage Diseases
- (2019) Angela McCall et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1
- (2019) Ewelina Mamcarz et al. NEW ENGLAND JOURNAL OF MEDICINE
- Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model
- (2019) Toshio Miki et al. Stem Cells International
- Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease
- (2019) Jacob T. Cain et al. MOLECULAR THERAPY
- Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA
- (2019) Stuart M. Ellison et al. Molecular Therapy-Methods & Clinical Development
- Adeno-associated virus in the liver: natural history and consequences in tumour development
- (2019) Tiziana La Bella et al. GUT
- Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease
- (2019) Sophia-Martha kleine Holthaus et al. HUMAN MOLECULAR GENETICS
- Liver-Directed AAV Gene Therapy for Gaucher Disease
- (2019) Carlos J Miranda et al. BLOOD
- A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna
- (2019) Toloo Taghian et al. MOLECULAR THERAPY
- Search-and-replace genome editing without double-strand breaks or donor DNA
- (2019) Andrew V. Anzalone et al. NATURE
- Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
- (2019) Natalia Gomez-Ospina et al. Nature Communications
- Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism
- (2019) Emily Y. Tan et al. Frontiers in Pediatrics
- Transduction Efficiency of Adeno-Associated Virus Serotypes After Local Injection in Mouse and Human Skeletal Muscle
- (2019) Laura Muraine et al. HUMAN GENE THERAPY
- Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes
- (2019) Giulia Massaro et al. HUMAN MOLECULAR GENETICS
- Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA
- (2019) Nicolina Cristina Sorrentino et al. Molecular Therapy-Methods & Clinical Development
- AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains
- (2019) Michaël Hocquemiller et al. Molecular Therapy-Methods & Clinical Development
- Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy
- (2018) Valeria Calbi et al. BONE MARROW TRANSPLANTATION
- A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
- (2018) Julie Tordo et al. BRAIN
- Main steps in DNA double-strand break repair: an introduction to homologous recombination and related processes
- (2018) Lepakshi Ranjha et al. CHROMOSOMA
- Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms
- (2018) Hélène FE Gleitz et al. EMBO Molecular Medicine
- Gene editing of MPS I human fibroblasts by co-delivery of a CRISPR/Cas9 plasmid and a donor oligonucleotide using nanoemulsions as nonviral carriers
- (2018) Roselena Silvestri Schuh et al. EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
- Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
- (2018) Christian Hinderer et al. HUMAN GENE THERAPY
- Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals
- (2018) Christian Hinderer et al. HUMAN GENE THERAPY
- Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay–Sachs Disease
- (2018) Heather L. Gray-Edwards et al. HUMAN GENE THERAPY
- Age-related seroprevalence of antibodies against AAV-LK03 in a UK population cohort
- (2018) Dany Perocheau et al. HUMAN GENE THERAPY
- AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease
- (2018) Michael P Hughes et al. HUMAN MOLECULAR GENETICS
- α-Synuclein accumulation and GBA deficiency due to L444P GBA mutation contributes to MPTP-induced parkinsonism
- (2018) Seung Pil Yun et al. Molecular Neurodegeneration
- Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing
- (2018) Kanut Laoharawee et al. MOLECULAR THERAPY
- Longitudinal in vivo monitoring of CNS demonstrates the efficacy of gene therapy in a sheep model of CLN5 Batten disease
- (2018) Nadia L. Mitchell et al. MOLECULAR THERAPY
- Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity
- (2018) Nicole K. Paulk et al. MOLECULAR THERAPY
- Prevention of Photoreceptor Cell Loss in a Cln6 nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells
- (2018) Sophia-Martha kleine Holthaus et al. MOLECULAR THERAPY
- Fetal gene therapy for neurodegenerative disease of infants
- (2018) Giulia Massaro et al. NATURE MEDICINE
- Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia
- (2018) Shannon L. Maude et al. NEW ENGLAND JOURNAL OF MEDICINE
- Standardized method for intra-cisterna magna delivery under fluoroscopic guidance in nonhuman primates
- (2018) Nathan Katz et al. Human Gene Therapy Methods
- Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques
- (2018) Juliette Hordeaux et al. Molecular Therapy-Methods & Clinical Development
- Emerging Issues in AAV-Mediated In Vivo Gene Therapy
- (2018) Pasqualina Colella et al. Molecular Therapy-Methods & Clinical Development
- Delivery of Gba Gene Using AAV9 Vector Therapy as a Treatment Strategy in Mouse Models of Gaucher disease
- (2018) Sichen Du et al. HUMAN GENE THERAPY
- In Vivo Genome Editing as a Therapeutic Approach
- (2018) Beatrice Ho et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Clinical challenges and future therapeutic approaches for neuronal ceroid lipofuscinosis
- (2018) Sara E Mole et al. LANCET NEUROLOGY
- ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome
- (2018) Li Ou et al. MOLECULAR THERAPY
- Base editing: precision chemistry on the genome and transcriptome of living cells
- (2018) Holly A. Rees et al. NATURE REVIEWS GENETICS
- Lysosomal storage diseases
- (2018) Frances M. Platt et al. Nature Reviews Disease Primers
- Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates
- (2018) Kousaku Ohno et al. Molecular Therapy-Methods & Clinical Development
- Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe
- (2017) Nadia Belmatoug et al. European Journal of Internal Medicine
- Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
- (2017) Carles Roca et al. HUMAN MOLECULAR GENETICS
- Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
- (2017) Julien Baruteau et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy
- (2017) Conrad A. Vink et al. MOLECULAR THERAPY
- Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage
- (2017) Nicole M. Gaudelli et al. NATURE
- Non-homologous DNA end joining and alternative pathways to double-strand break repair
- (2017) Howard H. Y. Chang et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
- (2017) Lindsey A. George et al. NEW ENGLAND JOURNAL OF MEDICINE
- Synergistic effects of treating the spinal cord and brain in CLN1 disease
- (2017) Charles Shyng et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
- (2017) Francesco Puzzo et al. Science Translational Medicine
- Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
- (2017) Giulia Schiroli et al. Science Translational Medicine
- Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
- (2017) Rita Ferla et al. Molecular Therapy-Methods & Clinical Development
- Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction
- (2017) Sang-oh Han et al. Molecular Therapy-Methods & Clinical Development
- Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease
- (2017) J. Hordeaux et al. Acta Neuropathologica Communications
- Migalastat: First Global Approval
- (2016) Anthony Markham DRUGS
- Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2Gangliosidosis in Sandhoff Mice
- (2016) Karlaina J.L. Osmon et al. HUMAN GENE THERAPY
- Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
- (2016) Subha Karumuthil-Melethil et al. HUMAN GENE THERAPY
- A Preclinical Study Evaluating AAVrh10-Based Gene Therapy for Sanfilippo Syndrome
- (2016) Leanne K. Winner et al. HUMAN GENE THERAPY
- Adeno-Associated Virus Gene Therapy for Liver Disease
- (2016) Lisa M. Kattenhorn et al. HUMAN GENE THERAPY
- Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
- (2016) Randy J. Chandler et al. HUMAN MOLECULAR GENETICS
- The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders
- (2016) A. Broomfield et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
- (2016) Derralynn A Hughes et al. JOURNAL OF MEDICAL GENETICS
- Coxsackievirus Adenovirus Receptor Loss Impairs Adult Neurogenesis, Synapse Content, and Hippocampus Plasticity
- (2016) C. Zussy et al. JOURNAL OF NEUROSCIENCE
- Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant
- (2016) Subha Karumuthil-Melethil et al. JOURNAL OF NEUROSCIENCE RESEARCH
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States
- (2016) Manisha Balwani et al. MOLECULAR GENETICS AND METABOLISM
- Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model
- (2016) Christian Hinderer et al. MOLECULAR GENETICS AND METABOLISM
- Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1
- (2016) Sea Young Yoon et al. MOLECULAR THERAPY
- 605. Cerebrospinal Fluid for Delivery of AAV Gene Therapy in GM1 Gangliosidosis
- (2016) Amanda L. Gross et al. MOLECULAR THERAPY
- Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII
- (2016) Brittney L Gurda et al. MOLECULAR THERAPY
- CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
- (2016) Daniel P. Dever et al. NATURE
- Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
- (2016) Alexis C. Komor et al. NATURE
- Origin, fate and dynamics of macrophages at central nervous system interfaces
- (2016) Tobias Goldmann et al. NATURE IMMUNOLOGY
- Cerebellomedullary Cistern Delivery for AAV-Based Gene Therapy: A Technical Note for Nonhuman Primates
- (2016) Lluis Samaranch et al. Human Gene Therapy Methods
- 604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I
- (2016) MOLECULAR THERAPY
- Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
- (2015) Adrian G. Todd et al. ANNALS OF NEUROLOGY
- Lysosomal Storage Diseases: From Pathophysiology to Therapy
- (2015) Giancarlo Parenti et al. Annual Review of Medicine
- AAV-Mediated Gene Delivery in a Feline Model of Sandhoff Disease Corrects Lysosomal Storage in the Central Nervous System
- (2015) Hannah E. Rockwell et al. ASN Neuro
- In vivo genome editing of the albumin locus as a platform for protein replacement therapy
- (2015) R. Sharma et al. BLOOD
- Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study
- (2015) M. Aldenhoven et al. BLOOD
- Organellar channels and transporters
- (2015) Haoxing Xu et al. CELL CALCIUM
- Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II
- (2015) Taichi Wakabayashi et al. HUMAN GENE THERAPY
- Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan
- (2015) Cara M. Weismann et al. HUMAN MOLECULAR GENETICS
- Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
- (2015) Randy J. Chandler et al. JOURNAL OF CLINICAL INVESTIGATION
- Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy
- (2015) Heather L. Gray-Edwards et al. MOLECULAR GENETICS AND METABOLISM
- Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features
- (2015) Mohammad A. Rafi et al. MOLECULAR GENETICS AND METABOLISM
- Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice
- (2015) F Jason Duncan et al. MOLECULAR THERAPY
- Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates
- (2015) Christian Hinderer et al. MOLECULAR THERAPY
- Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates
- (2015) Jagdeep S Walia et al. MOLECULAR THERAPY
- Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
- (2015) Giancarlo Parenti et al. MOLECULAR THERAPY
- Development of an intein-mediated split–Cas9 system for gene therapy
- (2015) Dong-Jiunn Jeffery Truong et al. NUCLEIC ACIDS RESEARCH
- Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C
- (2015) Simona Fecarotta et al. Orphanet Journal of Rare Diseases
- Adoptive cell transfer as personalized immunotherapy for human cancer
- (2015) S. A. Rosenberg et al. SCIENCE
- AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease
- (2015) Martin L. Katz et al. Science Translational Medicine
- Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector
- (2015) Kohei Hironaka et al. Scientific Reports
- Adoptive Immunotherapy for Cancer or Viruses
- (2014) Marcela V. Maus et al. Annual Review of Immunology
- TALEN-Mediated Generation and Genetic Correction of Disease-Specific Human Induced Pluripotent Stem Cells
- (2014) Sivaprakash Ramalingam et al. CURRENT GENE THERAPY
- Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
- (2014) Balaji Balakrishnan et al. CURRENT GENE THERAPY
- Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector
- (2014) N Miyake et al. GENE THERAPY
- Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy
- (2014) Albert Ribera et al. HUMAN MOLECULAR GENETICS
- Taliglucerase alfa: An enzyme replacement therapy using plant cell expression technology
- (2014) Gregory A. Grabowski et al. MOLECULAR GENETICS AND METABOLISM
- Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I
- (2014) Christian Hinderer et al. MOLECULAR THERAPY
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- (2014) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
- (2014) C. Hinderer et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Sustained Normalization of Neurological Disease after Intracranial Gene Therapy in a Feline Model
- (2014) V. J. McCurdy et al. Science Translational Medicine
- Targeted gene transfer into ependymal cells through intraventricular injection of AAV1 vector and long-term enzyme replacement via the CSF
- (2014) Yoshiyuki Yamazaki et al. Scientific Reports
- Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
- (2014) Gensheng Wang et al. Molecular Therapy-Methods & Clinical Development
- Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna
- (2014) Christian Hinderer et al. Molecular Therapy-Methods & Clinical Development
- Immune responses to AAV vectors: overcoming barriers to successful gene therapy
- (2013) F. Mingozzi et al. BLOOD
- Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates
- (2013) S J Gray et al. GENE THERAPY
- Central Nervous System Delivery of Helper-Dependent Canine Adenovirus Corrects Neuropathology and Behavior in Mucopolysaccharidosis Type VII Mice
- (2013) Lorena Ariza et al. HUMAN GENE THERAPY
- Partial Correction of the CNS Lysosomal Storage Defect in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis by Neonatal CNS Administration of an Adeno-Associated Virus Serotype rh.10 Vector Expressing the Human CLN3 Gene
- (2013) Dolan Sondhi et al. HUMAN GENE THERAPY
- Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy
- (2013) Virginia Haurigot et al. JOURNAL OF CLINICAL INVESTIGATION
- Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy
- (2013) Allison M Bradbury et al. MOLECULAR THERAPY
- Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease
- (2013) Ana Sergijenko et al. MOLECULAR THERAPY
- Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease
- (2013) Darin J Falk et al. MOLECULAR THERAPY
- Corrective GUSB Transfer to the Canine Mucopolysaccharidosis VII Brain
- (2013) Aurelie Cubizolle et al. MOLECULAR THERAPY
- Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice
- (2013) Mai K ElMallah et al. MOLECULAR THERAPY
- Lysosomal storage diseases—the horizon expands
- (2013) Rose-Mary Naaman Boustany Nature Reviews Neurology
- Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease
- (2013) Christopher G. Janson et al. NEUROSURGERY
- Cost-effectiveness of enzyme replacement therapy for Fabry disease
- (2013) Saskia M Rombach et al. Orphanet Journal of Rare Diseases
- Bone Marrow Cell Recruitment to the Brain in the Absence of Irradiation or Parabiosis Bias
- (2013) Katrin Kierdorf et al. PLoS One
- Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood
- (2013) Bartholomew J. Naughton et al. PLoS One
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges
- (2012) R.J. Desnick et al. Annual Review of Genomics and Human Genetics
- Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer
- (2012) G. Buchlis et al. BLOOD
- Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice
- (2012) Françoise Piguet et al. HUMAN GENE THERAPY
- Correction of Pathological Accumulation of Glycosaminoglycans in Central Nervous System and Peripheral Tissues of MPSIIIA Mice Through Systemic AAV9 Gene Transfer
- (2012) Albert Ruzo et al. HUMAN GENE THERAPY
- Sialic Acid Deposition Impairs the Utility of AAV9, but Not Peptide-modified AAVs for Brain Gene Therapy in a Mouse Model of Lysosomal Storage Disease
- (2012) Yong Hong Chen et al. MOLECULAR THERAPY
- Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice
- (2012) Alexander Langford-Smith et al. MOLECULAR THERAPY
- The AAV Vector Toolkit: Poised at the Clinical Crossroads
- (2012) Aravind Asokan et al. MOLECULAR THERAPY
- Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis
- (2012) Frank Harrison et al. MOLECULAR THERAPY
- Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation
- (2012) A. Capotondo et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
- (2012) M. Jinek et al. SCIENCE
- TAL effectors are remote controls for gene activation
- (2011) Heidi Scholze et al. CURRENT OPINION IN MICROBIOLOGY
- Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
- (2011) T Federici et al. GENE THERAPY
- Comparison of Adeno-Associated Viral Vector Serotypes for Spinal Cord and Motor Neuron Gene Delivery
- (2011) Brooke R. Snyder et al. HUMAN GENE THERAPY
- Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
- (2011) Lluis Samaranch et al. HUMAN GENE THERAPY
- Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
- (2011) Timothy J. Sargeant et al. HUMAN MOLECULAR GENETICS
- The cellular pathology of lysosomal diseases
- (2011) Timothy M Cox et al. JOURNAL OF PATHOLOGY
- Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
- (2011) Steven J Gray et al. MOLECULAR THERAPY
- Efficacy of a Combined Intracerebral and Systemic Gene Delivery Approach for the Treatment of a Severe Lysosomal Storage Disorder
- (2011) Carmine Spampanato et al. MOLECULAR THERAPY
- Ex vivo gene transfer and correction for cell-based therapies
- (2011) Luigi Naldini NATURE REVIEWS GENETICS
- Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I
- (2011) Daniel A. Wolf et al. NEUROBIOLOGY OF DISEASE
- Enzyme Replacement Therapy and/or Hematopoietic Stem Cell Transplantation at diagnosis in patients with Mucopolysaccharidosis type I: results of a European consensus procedure
- (2011) Minke H de Ru et al. Orphanet Journal of Rare Diseases
- The Mechanism of Double-Strand DNA Break Repair by the Nonhomologous DNA End-Joining Pathway
- (2010) Michael R. Lieber Annual Review of Biochemistry
- Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience
- (2010) A. Zimran et al. BLOOD
- Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype
- (2010) N. P. van Til et al. BLOOD
- A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells
- (2010) S. Zhou et al. BLOOD
- Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model
- (2010) I. Visigalli et al. BLOOD
- Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
- (2010) M. Christian et al. GENETICS
- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
- (2010) Sylvie Boutin et al. HUMAN GENE THERAPY
- Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer
- (2010) Chantelle McIntyre et al. JOURNAL OF GENE MEDICINE
- Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII
- (2010) Julie Bielicki et al. MOLECULAR GENETICS AND METABOLISM
- SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors
- (2010) Adeline A. Lau et al. MOLECULAR GENETICS AND METABOLISM
- Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease
- (2010) Cathryn S Mah et al. MOLECULAR THERAPY
- Therapeutic Efficacy of Bone Marrow Transplant, Intracranial AAV-mediated Gene Therapy, or Both in the Mouse Model of MPS IIIB
- (2010) Coy D Heldermon et al. MOLECULAR THERAPY
- AAV Vectors Avoid Inflammatory Signals Necessary to Render Transduced Hepatocyte Targets for Destructive T Cells
- (2010) Suryanarayan Somanathan et al. MOLECULAR THERAPY
- Safe, Efficient, and Reproducible Gene Therapy of the Brain in the Dog Models of Sanfilippo and Hurler Syndromes
- (2010) N Matthew Ellinwood et al. MOLECULAR THERAPY
- Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer
- (2010) Gabriella Cotugno et al. MOLECULAR THERAPY
- Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
- (2010) Sung-Chul Jung et al. MOLECULES AND CELLS
- A TALE nuclease architecture for efficient genome editing
- (2010) Jeffrey C Miller et al. NATURE BIOTECHNOLOGY
- AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival
- (2010) Rena C. Baek et al. PLoS One
- An Update on Canine Adenovirus Type 2 and Its Vectors
- (2010) Thierry Bru et al. Viruses-Basel
- IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice
- (2009) Vinicia Assunta Polito et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction
- (2009) S O Kyosen et al. GENE THERAPY
- Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice
- (2009) D M McCarty et al. GENE THERAPY
- The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy
- (2009) Eugenio Montini et al. JOURNAL OF CLINICAL INVESTIGATION
- Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α-glucosidase in Pompe disease
- (2009) Gaëlle Douillard-Guilloux et al. JOURNAL OF GENE MEDICINE
- A Plant-Derived Recombinant Human Glucocerebrosidase Enzyme—A Preclinical and Phase I Investigation
- (2009) David Aviezer et al. PLoS One
- Substrate reduction therapy
- (2008) Frances M Platt et al. ACTA PAEDIATRICA
- Treating lysosomal storage disorders: Current practice and future prospects
- (2008) Frances M. Platt et al. BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH
- The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation
- (2008) Mieke Aldenhoven et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Gene Therapy Using Adeno-Associated Virus Vectors
- (2008) S. Daya et al. CLINICAL MICROBIOLOGY REVIEWS
- Heat shock response relieves ER stress
- (2008) Yu Liu et al. EMBO JOURNAL
- Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA
- (2008) Chantelle McIntyre et al. MOLECULAR GENETICS AND METABOLISM
- Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors
- (2008) Sharon C Cunningham et al. MOLECULAR THERAPY
- Successful Low-Risk Hematopoietic Cell Therapy in a Mouse Model of Type 1 Gaucher Disease
- (2008) Ida Berglin Enquist et al. STEM CELLS
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