The potential of gene therapy for mucopolysaccharidosis type I

Viral Delivery Systems for CRISPR

(2019) Christine Xu et al.   Viruses-Basel

Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors

(2019) Hildegard Büning et al.   Molecular Therapy-Methods & Clinical Development

EMPOWERS: A phase 1/2 clinical trial of SB-318 ZFN-mediated in vivo human genome editing for treatment of MPS I (Hurler syndrome)

(2019) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Polymeric Nanoparticles in Gene Therapy: New Avenues of Design and Optimization for Delivery Applications

(2019) Raj Rai et al.   Polymers

Cas9 Ribonucleoprotein Complex Delivery: Methods and Applications for Neuroinflammation

(2019) Lee A. Campbell et al.   Journal of Neuroimmune Pharmacology

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

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases

(2018) Rebecca J. Holley et al.   ACS Chemical Neuroscience

In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system

(2018) Roselena Silvestri Schuh et al.   JOURNAL OF CONTROLLED RELEASE

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

(2018) Li Ou et al.   MOLECULAR THERAPY

Sangamo's landmark genome editing trial gets mixed reception

(2018) Cormac Sheridan   NATURE BIOTECHNOLOGY

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

(2017) Lalitha R. Belur et al.   HUMAN GENE THERAPY

Open issues in Mucopolysaccharidosis type I-Hurler

(2017) Rossella Parini et al.   Orphanet Journal of Rare Diseases

Gene editing for cell engineering: trends and applications

(2016) Sanjeev K. Gupta et al.   CRITICAL REVIEWS IN BIOTECHNOLOGY

Genome engineering tools for building cellular models of disease

(2016) Jennie Lin et al.   FEBS Journal

Preclinical Testing of the Safety and Tolerability of Lentiviral Vector–Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

(2016) Ilaria Visigalli et al.   HUMAN GENE THERAPY

ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus

(2016) Li Ou et al.   MOLECULAR GENETICS AND METABOLISM

Genome-Editing Technologies: Principles and Applications

(2016) Thomas Gaj et al.   Cold Spring Harbor Perspectives in Biology

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

Gene therapy for lysosomal storage disorders: a good start

(2015) Alessandra Biffi   HUMAN MOLECULAR GENETICS

Current and future delivery systems for engineered nucleases: ZFN, TALEN and RGEN

(2015) Qurrat Ul Ain et al.   JOURNAL OF CONTROLLED RELEASE

α-l-iduronidase gene-based therapy using the phiC31 system to treat mucopolysaccharidose type I mice

(2015) Roberta Sessa Stilhano et al.   JOURNAL OF GENE MEDICINE

479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease

(2015) Thomas Wechsler et al.   MOLECULAR THERAPY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

Lysosomal storage diseases as disorders of autophagy

(2014) Carmine Settembre et al.   Autophagy

Development and Applications of CRISPR-Cas9 for Genome Engineering

(2014) Patrick D. Hsu et al.   CELL

Clinical potential of gene therapy: towards meeting the demand

(2014) J. L. Macpherson et al.   INTERNAL MEDICINE JOURNAL

Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase

(2014) Salim S El-Amouri et al.   MOLECULAR THERAPY

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

(2014) Christian Hinderer et al.   MOLECULAR THERAPY

Mesenchymal Stem Cells Do Not Prevent Antibody Responses against Human α-L-Iduronidase when Used to Treat Mucopolysaccharidosis Type I

(2014) Priscila Keiko Matsumoto Martin et al.   PLoS One

Early initiation of enzyme replacement therapy for the mucopolysaccharidoses

(2013) Joseph Muenzer   MOLECULAR GENETICS AND METABOLISM

Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease

(2013) Christopher G. Janson et al.   NEUROSURGERY

Efficacy and Safety of Sleeping Beauty Transposon-Mediated Gene Transfer in Preclinical Animal Studies

(2012) Perry B. Hackett et al.   CURRENT GENE THERAPY

Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits

(2012) Guilherme Baldo et al.   JOURNAL OF INHERITED METABOLIC DISEASE

In vivo genome editing using a high-efficiency TALEN system

(2012) Victoria M. Bedell et al.   NATURE

Genome Engineering With Zinc-Finger Nucleases

(2011) Dana Carroll   GENETICS

The Sleeping Beauty transposon system: a non-viral vector for gene therapy

(2011) E. L. Aronovich et al.   HUMAN MOLECULAR 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

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

Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-L-Iduronidase in Mice With Mucopolysaccharidosis Type I

(2010) Mark J Osborn et al.   MOLECULAR THERAPY

AAV1-, AAV2- and AAV5-Mediated human α-Iduronidase Gene Transfer In The Brain of Nonhuman Primate: Vector Diffusion and Bio Distribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System

(2009) Elena L Aronovich et al.   MOLECULAR THERAPY

A Self-inactivating γ-Retroviral Vector Reduces Manifestations of Mucopolysaccharidosis I in Mice

(2009) Jason A Metcalf et al.   MOLECULAR THERAPY

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

(2009) D. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sorting of lysosomal proteins

(2008) Thomas Braulke et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

(2008) Ramin Sedaghat Herati et al.   JOURNAL OF GENE MEDICINE

Mucopolysaccharidosis I: Management and Treatment Guidelines

(2008) J. Muenzer et al.   PEDIATRICS