Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities

RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestations

(2021) Kirsten Cowley et al.   MOLECULAR GENETICS AND METABOLISM

RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial

(2021) Marie-Laure Nevoret et al.   MOLECULAR GENETICS AND METABOLISM

Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II

(2021) Joo-Hyun Seo et al.   Molecular Therapy-Methods & Clinical Development

Novel Enzyme Replacement Therapies for Neuropathic Mucopolysaccharidoses

(2020) Yuji Sato et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Genome Editing for Mucopolysaccharidoses

(2020) Edina Poletto et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Mucopolysaccharidosis Type II: One Hundred Years of Research, Diagnosis, and Treatment

(2020) Francesca D’Avanzo et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Setup and Validation of a Targeted Next-Generation Sequencing Approach for the Diagnosis of Lysosomal Storage Disorders

(2020) Alessandra Zanetti et al.   JOURNAL OF MOLECULAR DIAGNOSTICS

Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG)

(2020) Kim L. McBride et al.   GENETICS IN MEDICINE

A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II

(2020) Torayuki Okuyama et al.   MOLECULAR THERAPY

Natural history of cognitive development in neuronopathic mucopolysaccharidosis type II (Hunter syndrome): Contribution of genotype to cognitive developmental course

(2020) Joo-Hyun Seo et al.   Molecular Genetics and Metabolism Reports

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

Mucopolysaccharidoses I and II: Brief Review of Therapeutic Options and Supportive/Palliative Therapies

(2020) Haiyan Nan et al.   Biomed Research International

Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

(2020) Miho Wada et al.   Molecular Therapy-Methods & Clinical Development

Non-clinical evaluation of a blood-brain barrier-penetrating enzyme for the treatment of mucopolysaccharidosis type I

(2019) Sachiho Kida et al.   MOLECULAR GENETICS AND METABOLISM

Phenotype-oriented NGS panels for mucopolysaccharidoses: Validation and potential use in the diagnostic flowchart

(2019) Ana Carolina Brusius-Facchin et al.   GENETICS AND MOLECULAR BIOLOGY

Therapeutic Options for Mucopolysaccharidoses: Current and Emerging Treatments

(2019) Kazuki Sawamoto et al.   DRUGS

Autophagy in the Central Nervous System and Effects of Chloroquine in Mucopolysaccharidosis Type II Mice

(2019) Mitsuyo Maeda et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic

(2018) Wei-Wei Zhang et al.   HUMAN GENE THERAPY

FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII)

(2018) Stefania Bellesso et al.   HUMAN MOLECULAR GENETICS

The efficacy of intracerebroventricular idursulfase-beta enzyme replacement therapy in mucopolysaccharidosis II murine model: heparan sulfate in cerebrospinal fluid as a clinical biomarker of neuropathology

(2018) Young Bae Sohn et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Clinical use of lentiviral vectors

(2018) Michael C. Milone et al.   LEUKEMIA

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

First in vivo human genome editing trial

(2018)   NATURE BIOTECHNOLOGY

Gene Editing on Center Stage

(2018) Rasmus O. Bak et al.   TRENDS IN GENETICS

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

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

Sangamo's landmark genome editing trial gets mixed reception

(2018) Cormac Sheridan   NATURE BIOTECHNOLOGY

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene Transfer

(2017) Kanut Laoharawee et al.   HUMAN GENE THERAPY

Epidemiology of mucopolysaccharidoses

(2017) Shaukat A. Khan et al.   MOLECULAR GENETICS AND METABOLISM

Guanidinylated Neomycin Conjugation Enhances Intranasal Enzyme Replacement in the Brain

(2017) Wenyong Tong et al.   MOLECULAR THERAPY

Presentation and treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome)

(2017) Molly Stapleton et al.   Expert Opinion on Orphan Drugs

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

(2016) Christian Hinderer et al.   HUMAN GENE THERAPY

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Hematopoietic Stem Cell Gene Therapy Corrects Neuropathic Phenotype in Murine Model of Mucopolysaccharidosis Type II

(2015) Taichi Wakabayashi et al.   HUMAN GENE THERAPY

In vivo genome editing using Staphylococcus aureus Cas9

(2015) F. Ann Ran et al.   NATURE

Mucopolysaccharidosis type II in a female patient with a reciprocal X;9 translocation and skewed X chromosome inactivation

(2014) Fortunato Lonardo et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

(2014) Balaji Balakrishnan et al.   CURRENT GENE THERAPY

Long-term experience with enzyme replacement therapy (ERT) in MPS II patients with a severe phenotype: an international case series

(2014) Christina Lampe et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Gene therapy on the move

(2013) Kerstin B. Kaufmann et al.   EMBO Molecular Medicine

Mucopolysaccharidosis type II in females and response to enzyme replacement therapy

(2012) Agnieszka Jurecka et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Lentiviral vectors: basic to translational

(2012) Toshie Sakuma et al.   BIOCHEMICAL JOURNAL

Induced Pluripotent Stem Cells: Past, Present, and Future

(2012) Shinya Yamanaka   Cell Stem Cell

Mucopolysaccharidosis type II in a female carrying a heterozygous stop mutation of the iduronate-2-sulfatase gene and showing a skewed X chromosome inactivation

(2012) Raul E. Piña-Aguilar et al.   European Journal of Medical Genetics

Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II

(2012) Takashi Higuchi et al.   MOLECULAR GENETICS AND METABOLISM

Patterns of scAAV Vector Insertion Associated With Oncogenic Events in a Mouse Model for Genotoxicity

(2012) Lucia E Rosas et al.   MOLECULAR THERAPY

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

Glybera and the future of gene therapy in the European Union

(2012) Norman Miller   NATURE REVIEWS DRUG DISCOVERY

Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

(2011) Roberto Calcedo et al.   Clinical and Vaccine Immunology

A mother and daughter with the p.R443X mutation of mucopolysaccharidosis type II: Genotype and phenotype analysis

(2010) Young Bae Sohn et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Female Hunter syndrome caused by a single mutation and familial XCI skewing: implications for other X-linked disorders

(2010) A Kloska et al.   CLINICAL 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

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

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation

(2009) Ken Araya et al.   MOLECULAR GENETICS AND METABOLISM

Gene therapy of Hunter syndrome: Evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS)

(2008) A. Friso et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Adeno-associated virus integration: virus versus vector

(2008) R H Smith   GENE THERAPY