A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I-Reference-Cited by-同舟云学术

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

Published:2020-06 Issue:6 Volume:28 Page:1442-1454
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Ou Li,Przybilla Michael J.,Ahlat Ozan,Kim Sarah,Overn Paula,Jarnes Jeanine,O’Sullivan M. Gerard,Whitley Chester B.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference64 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Reversal of clinical features of Hurler’s disease and biochemical improvement after treatment by bone-marrow transplantation;Hobbs;Lancet,1981

3. Bone marrow transplantation for Hurler syndrome: assessment of metabolic correction;Whitley;Birth Defects Orig. Artic. Ser.,1986

4. Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison;Eisengart;Genet. Med.,2018

5. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-l-iduronidase (laronidase);Wraith;J. Pediatr.,2004

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