Clinical development on the frontier: gene therapy for duchenne muscular dystrophy-Reference-Cited by-同舟云学术

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Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

Published:2020-02-12 Issue:3 Volume:20 Page:263-274
ISSN:1471-2598
Container-title:Expert Opinion on Biological Therapy
language:en
Short-container-title:Expert Opinion on Biological Therapy

Author:

Asher Damon R.¹,Thapa Khampaseuth¹,Dharia Sachi D.¹,Khan Navid¹,Potter Rachael A.¹,Rodino-Klapac Louise R.¹,Mendell Jerry R.²³

Affiliation:

1. Sarepta Therapeutics, Inc, Cambridge, Massachusetts, USA

2. Center for Gene Therapy, The Abigail Wexner, Research Institute at Nationwide Children’s Hospital, Columbus, Ohio, USA

3. Department of Pediatrics and Neurology, The Ohio State University, Columbus, Ohio, USA

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology

Link

https://www.tandfonline.com/doi/pdf/10.1080/14712598.2020.1725469

Reference79 articles.

1. Gene therapy: progress and predictions

2. Gene Therapy for Human Genetic Disease?

3. Gene Therapy Death Prompts Review of Adenovirus Vector

4. Progress and problems with the use of viral vectors for gene therapy

5. A Serious Adverse Event after Successful Gene Therapy for X-Linked Severe Combined Immunodeficiency

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1. Correction of exon 2, exon 2–9 and exons 8–9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system;Scientific Reports;2024-09-11

2. Poly(vinyl alcohol) (PVA) for gene delivery: a systematic review;Journal of Drug Delivery Science and Technology;2024-09

3. Subspecialty Health Care Utilization in Pediatric Patients With Muscular Dystrophy in the United States;Neurology Clinical Practice;2024-08

4. Molecular and Biochemical Therapeutic Strategies for Duchenne Muscular Dystrophy;Neurology International;2024-07-05

5. Long-term clinical follow-up of a family with Becker muscular dystrophy associated with a large deletion in the DMD gene;Neuromuscular Disorders;2024-06

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