Mucopolysaccharidoses type I gene therapy

Immunotherapy to get on point with base editing

(2021) Jennifer A. Harbottle   DRUG DISCOVERY TODAY

Addressing the dark matter of gene therapy: technical and ethical barriers to clinical application

(2021) Kateryna Kratzer et al.   HUMAN GENETICS

Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing

(2021) Mitchell L. Leibowitz et al.   NATURE GENETICS

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I

(2020) Li Ou et al.   MOLECULAR THERAPY

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

(2020) Juliette Hordeaux et al.   HUMAN GENE THERAPY

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

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

(2020) Giang N. Nguyen et al.   NATURE BIOTECHNOLOGY

To win at gene therapy, companies pick viruses with production credentials

(2019) Cormac Sheridan   NATURE BIOTECHNOLOGY

Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

(2019) Calvin J. Stephens et al.   JOURNAL OF CONTROLLED RELEASE

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

(2019) Roselena Silvestri Schuh et al.   GENE THERAPY

Gene therapy for Mucopolysaccharidoses

(2018) Kazuki Sawamoto et al.   MOLECULAR GENETICS AND METABOLISM

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

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

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

Nasal Administration of Cationic Nanoemulsions as Nucleic Acids Delivery Systems Aiming at Mucopolysaccharidosis Type I Gene Therapy

(2018) Roselena Silvestri Schuh et al.   PHARMACEUTICAL RESEARCH

Deamidation of Amino Acids on the Surface of Adeno-Associated Virus Capsids Leads to Charge Heterogeneity and Altered Vector Function

(2018) April R. Giles et al.   MOLECULAR THERAPY

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

(2018) Li Ou et al.   MOLECULAR THERAPY

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

Cationic nanoemulsions as nucleic acids delivery systems

(2017) Helder Ferreira Teixeira et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

Engineered CRISPR Systems for Next Generation Gene Therapies

(2017) Michael Pineda et al.   ACS Synthetic Biology

Long Terminal Repeats: From Parasitic Elements to Building Blocks of the Transcriptional Regulatory Repertoire

(2016) Peter J. Thompson et al.   MOLECULAR CELL

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

Current Progress in Therapeutic Gene Editing for Monogenic Diseases

(2016) Versha Prakash et al.   MOLECULAR THERAPY

AAV Gene Therapy for MPS1-associated Corneal Blindness

(2016) Melisa Vance et al.   Scientific Reports

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates

(2015) Christian Hinderer et al.   MOLECULAR THERAPY

Adenovirus Vectors for Gene Therapy, Vaccination and Cancer Gene Therapy

(2014) William Wold et al.   CURRENT GENE THERAPY

The myeloid-binding peptide adenoviral vector enables multi-organ vascular endothelial gene targeting

(2014) Zhi Hong Lu et al.   LABORATORY INVESTIGATION

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

(2014) Christian Hinderer et al.   MOLECULAR THERAPY

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

Diagnosis and treatment trends in mucopolysaccharidosis I: findings from the MPS I Registry

(2012) Kristin D’Aco et al.   EUROPEAN JOURNAL OF PEDIATRICS

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

Lentiviral Vectors in Gene Therapy: Their Current Status and Future Potential

(2010) David Escors et al.   ARCHIVUM IMMUNOLOGIAE ET THERAPIAE EXPERIMENTALIS

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

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

Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

(2009) James M. Wilson   MOLECULAR GENETICS AND METABOLISM

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

(2009) Jason A Metcalf et al.   MOLECULAR THERAPY

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

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

(2008) David Moore et al.   Orphanet Journal of Rare Diseases