Genetic therapy for the nervous system

Regeneration of the MPTP-Lesioned Dopaminergic System after Convection-Enhanced Delivery of AAV2-GDNF

(2013) A. P. Kells et al.   JOURNAL OF NEUROSCIENCE

Parkinson's Disease: Genetics and Pathogenesis

(2011) Joshua M. Shulman et al.   Annual Review of Pathology-Mechanisms of Disease

The ABCG2 resistance network of glioblastoma

(2011) Anne-Marie Bleau et al.   CELL CYCLE

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

The Promise of MicroRNA Replacement Therapy

(2010) A. G. Bader et al.   CANCER RESEARCH

Oncolytic viruses as experimental treatments for malignant gliomas: Using a scourge to treat a devil

(2010) Franz J. Zemp et al.   CYTOKINE & GROWTH FACTOR REVIEWS

Preparing an oncolytic poliovirus recombinant for clinical application against glioblastoma multiforme

(2010) Christian Goetz et al.   CYTOKINE & GROWTH FACTOR REVIEWS

Lack of humoral immune response to the tetracycline (Tet) activator in rats injected intracranially with Tet-off rAAV vectors

(2010) Y Han et al.   GENE THERAPY

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

Magnetic Resonance Imaging-Guided Delivery of Adeno-Associated Virus Type 2 to the Primate Brain for the Treatment of Lysosomal Storage Disorders

(2010) E. Aguilar Salegio et al.   HUMAN GENE THERAPY

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

(2010) Elisa Dominguez et al.   HUMAN MOLECULAR GENETICS

Spinal muscular atrophy: mechanisms and therapeutic strategies

(2010) C. L. Lorson et al.   HUMAN MOLECULAR GENETICS

Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

(2010) Annalisa Lattanzi et al.   HUMAN MOLECULAR GENETICS

Systemic combinatorial peptide selection yields a non-canonical iron-mimicry mechanism for targeting tumors in a mouse model of human glioblastoma

(2010) Fernanda I. Staquicini et al.   JOURNAL OF CLINICAL INVESTIGATION

Biopharmaceutical drug targeting to the brain

(2010) William M. Pardridge   JOURNAL OF DRUG TARGETING

Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy

(2010) T. H. Coady et al.   JOURNAL OF NEUROSCIENCE

An Engineered Zinc Finger Protein Activator of the Endogenous Glial Cell Line-Derived Neurotrophic Factor Gene Provides Functional Neuroprotection in a Rat Model of Parkinson's Disease

(2010) J. Laganiere et al.   JOURNAL OF NEUROSCIENCE

Trafficking CD11b-Positive Blood Cells Deliver Therapeutic Genes to the Brain of Amyloid-Depositing Transgenic Mice

(2010) L. Lebson et al.   JOURNAL OF NEUROSCIENCE

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

(2010) William J Marks et al.   LANCET NEUROLOGY

Robust In Vivo Transduction of Nervous System and Neural Stem Cells by Early Gestational Intra Amniotic Gene Transfer Using Lentiviral Vector

(2010) David H Stitelman et al.   MOLECULAR THERAPY

Lentivirus-mediated Reprogramming of Somatic Cells in the Absence of Transgenic Transcription Factors

(2010) Nicole M Kane et al.   MOLECULAR THERAPY

Aβ-directed Single-chain Antibody Delivery Via a Serotype-1 AAV Vector Improves Learning Behavior and Pathology in Alzheimer's Disease Mice

(2010) Deborah A Ryan et al.   MOLECULAR THERAPY

Toward Brain Tumor Gene Therapy Using Multipotent Mesenchymal Stromal Cell Vectors

(2010) Daniel Bexell et al.   MOLECULAR THERAPY

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

Efficacy and Safety/Toxicity Study of Recombinant Vaccinia Virus JX-594 in Two Immunocompetent Animal Models of Glioma

(2010) XueQing Lun et al.   MOLECULAR THERAPY

In Vivo RNAi-Mediated α-Synuclein Silencing Induces Nigrostriatal Degeneration

(2010) Oleg S Gorbatyuk et al.   MOLECULAR THERAPY

AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice

(2010) James C Dodge et al.   MOLECULAR THERAPY

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

(2010) Shin-ichi Muramatsu et al.   MOLECULAR THERAPY

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

(2010) Jun Xie et al.   MOLECULAR THERAPY

Pericytes regulate the blood–brain barrier

(2010) Annika Armulik et al.   NATURE

Mutations of optineurin in amyotrophic lateral sclerosis

(2010) Hirofumi Maruyama et al.   NATURE

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Efficient CNS gene delivery by intravenous injection

(2010) Jean-Pierre Louboutin et al.   NATURE METHODS

Increased Expression of α-Synuclein Reduces Neurotransmitter Release by Inhibiting Synaptic Vesicle Reclustering after Endocytosis

(2010) Venu M. Nemani et al.   NEURON

Misfolded Mutant SOD1 Directly Inhibits VDAC1 Conductance in a Mouse Model of Inherited ALS

(2010) Adrian Israelson et al.   NEURON

Genetic Animal Models of Parkinson's Disease

(2010) Ted M. Dawson et al.   NEURON

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mesenchymal Stem Cells Modified with a Single-Chain Antibody against EGFRvIII Successfully Inhibit the Growth of Human Xenograft Malignant Glioma

(2010) Irina V. Balyasnikova et al.   PLoS One

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

Gene therapy-mediated delivery of targeted cytotoxins for glioma therapeutics

(2010) M. Candolfi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In vivo electroporation of the central nervous system: A non-viral approach for targeted gene delivery

(2010) Jochen De Vry et al.   PROGRESS IN NEUROBIOLOGY

Human Induced Pluripotent Stem Cells Develop Teratoma More Efficiently and Faster Than Human Embryonic Stem Cells Regardless the Site of Injection

(2010) Ivan Gutierrez-Aranda et al.   STEM CELLS

Concise Review: Stem Cells As an Emerging Platform for Antibody Therapy of Cancer

(2010) Richard T. Frank et al.   STEM CELLS

Controlling brain tumor growth by intraventricular administration of an AAV vector encoding IFN-β

(2009) D H Meijer et al.   CANCER GENE THERAPY

Human Bone Marrow-Derived Mesenchymal Stem Cells for Intravascular Delivery of Oncolytic Adenovirus  24-RGD to Human Gliomas

(2009) R. L. Yong et al.   CANCER RESEARCH

Mediators of Glioblastoma Resistance and Invasion during Antivascular Endothelial Growth Factor Therapy

(2009) A. K. Lucio-Eterovic et al.   CLINICAL CANCER RESEARCH

Adenovirus Mediated Herpes Simplex Virus-Thymidine Kinase/Ganciclovir Gene Therapy for Resectable Malignant Glioma

(2009) Ann-Marie Maatta et al.   CURRENT GENE THERAPY

Specific targeted gene repair using single-stranded DNA oligonucleotides at an endogenous locus in mammalian cells uses homologous recombination

(2009) Jennifer McLachlan et al.   DNA REPAIR

Serotonin neuron-dependent and -independent reduction of dyskinesia by 5-HT1A and 5-HT1B receptor agonists in the rat Parkinson model

(2009) Ana Muñoz et al.   EXPERIMENTAL NEUROLOGY

Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors

(2009) A A Rahim et al.   GENE THERAPY

Targeting novel integrative nuclear FGFR1 signaling by nanoparticle-mediated gene transfer stimulates neurogenesis in the adult brain

(2009) Ewa K. Stachowiak et al.   Integrative Biology

Oligonucleotide-Mediated Survival of Motor Neuron Protein Expression in CNS Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy

(2009) J. H. Williams et al.   JOURNAL OF NEUROSCIENCE

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

An epidermal growth factor receptor variant III-targeted vaccine is safe and immunogenic in patients with glioblastoma multiforme

(2009) J. H. Sampson et al.   MOLECULAR CANCER THERAPEUTICS

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Physiological Transgene Regulation and Functional Complementation of a Neurological Disease Gene Deficiency in Neurons

(2009) Pier Paolo Peruzzi et al.   MOLECULAR THERAPY

Tight Long-term Dynamic Doxycycline Responsive Nigrostriatal GDNF Using a Single rAAV Vector

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

Directed Evolution of a Novel Adeno-associated Virus (AAV) Vector That Crosses the Seizure-compromised Blood–Brain Barrier (BBB)

(2009) Steven J Gray et al.   MOLECULAR THERAPY

Adeno-associated Virus Gene Therapy With Cholesterol 24-Hydroxylase Reduces the Amyloid Pathology Before or After the Onset of Amyloid Plaques in Mouse Models of Alzheimer's Disease

(2009) Eloise Hudry et al.   MOLECULAR THERAPY

Alnylam dealt blow

(2009) Asher Mullard   NATURE BIOTECHNOLOGY

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

(2009) Yong Hong Chen et al.   NATURE MEDICINE

Genetics of motor neuron disorders: new insights into pathogenic mechanisms

(2009) Patrick A. Dion et al.   NATURE REVIEWS GENETICS

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Hearing Improvement after Bevacizumab in Patients with Neurofibromatosis Type 2

(2009) Scott R. Plotkin et al.   NEW ENGLAND JOURNAL OF MEDICINE

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

(2009) L. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Directed evolution of adeno-associated virus to an infectious respiratory virus

(2009) K. J. D. A. Excoffon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mutations in FUS, an RNA Processing Protein, Cause Familial Amyotrophic Lateral Sclerosis Type 6

(2009) C. Vance et al.   SCIENCE

Mutations in the FUS/TLS Gene on Chromosome 16 Cause Familial Amyotrophic Lateral Sclerosis

(2009) T. J. Kwiatkowski et al.   SCIENCE

Prospects of stem cell therapy for replacing dopamine neurons in Parkinson's disease

(2009) Olle Lindvall et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Tumor-Targeted Interferon-α Delivery by Tie2-Expressing Monocytes Inhibits Tumor Growth and Metastasis

(2008) Michele De Palma et al.   CANCER CELL

Primary neural stem/progenitor cells expressing endostatin or cytochrome P450 for gene therapy of glioblastoma

(2008) A Lorico et al.   CANCER GENE THERAPY

Guidelines and Techniques for the Generation of Induced Pluripotent Stem Cells

(2008) Nimet Maherali et al.   Cell Stem Cell

Stem and progenitor cell-mediated tumor selective gene therapy

(2008) K S Aboody et al.   GENE THERAPY

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

(2008) Michael Chang et al.   MOLECULAR THERAPY

Advances in High-capacity Extrachromosomal Vector Technology: Episomal Maintenance, Vector Delivery, and Transgene Expression

(2008) Michele MP Lufino et al.   MOLECULAR THERAPY

Preventing Growth of Brain Tumors by Creating a Zone of Resistance

(2008) Casey A Maguire et al.   MOLECULAR THERAPY

Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity

(2008) James C Dodge et al.   MOLECULAR THERAPY

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY

DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

(2008) James T Koerber et al.   MOLECULAR THERAPY

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

(2008) Mark J Osborn et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

(2008) J. L. Eberling et al.   NEUROLOGY

Selective Gene Expression by Postnatal Electroporation during Olfactory Interneuron Neurogenesis

(2008) Alexander T. Chesler et al.   PLoS One

Reprogramming of murine and human somatic cells using a single polycistronic vector

(2008) B. W. Carey et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TDP-43 Mutations in Familial and Sporadic Amyotrophic Lateral Sclerosis

(2008) J. Sreedharan et al.   SCIENCE

Induced Pluripotent Stem Cell Generation Using a Single Lentiviral Stem Cell Cassette

(2008) Cesar A. Sommer et al.   STEM CELLS