Systemic Correction of Storage Disease in MPS I NOD/SCID Mice Using the Sleeping Beauty Transposon System-Reference-Cited by-同舟云学术

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

Published:2009-07 Issue:7 Volume:17 Page:1136-1144
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Aronovich Elena L,Bell Jason B,Khan Shaukat A,Belur Lalitha R,Gunther Roland,Koniar Brenda,Schachern Patricia A,Parker Josh B,Carlson Cathy S,Whitley Chester B,McIvor R Scott,Gupta Pankaj,Hackett Perry B

Publisher

Elsevier BV

Subject

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

Reference50 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Long-term outcome of Hurler syndrome following bone marrow transplantation;Whitley;Am J Med Genet,1993

3. Cell and gene-based therapies for the lysosomal storage diseases;Hodges;Curr Gene Ther,2006

4. Limitations of enzyme replacement therapy: current and future;Wraith;J Inherit Metab Dis,2006

5. Gene therapy for mucopolysaccharidosis;Ponder;Expert Opin Biol Ther,2007

Cited by 77 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice;Molecular Therapy - Methods & Clinical Development;2024-03

2. Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy;International Journal of Molecular Sciences;2024-01-17

3. MPSI Manifestations and Treatment Outcome: Skeletal Focus;International Journal of Molecular Sciences;2022-09-22

4. Clinical Development of Cell Therapies to Halt Lysosomal Storage Diseases: Results and Lessons Learned;Current Gene Therapy;2021-07-28

5. Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I;Frontiers in Molecular Neuroscience;2021-05-10