Correction of Methylmalonic AciduriaIn VivoUsing a Codon-Optimized Lentiviral Vector-Reference-Cited by-同舟云学术

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Correction of Methylmalonic AciduriaIn VivoUsing a Codon-Optimized Lentiviral Vector

Published:2014-06 Issue:6 Volume:25 Page:529-538
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Wong Edward S.Y.¹²,McIntyre Chantelle¹²,Peters Heidi L.³,Ranieri Enzo¹,Anson Donald S.¹²,Fletcher Janice M.¹²

Affiliation:

1. Genetics and Molecular Pathology, Women's and Children's Hospital, North Adelaide, SA 5006, Australia.

2. School of Paediatrics and Reproductive Health, University of Adelaide, North Adelaide, SA 5006, Australia.

3. Metabolic Research, Murdoch Children's Research Institute, Royal Children's Hospital, Parkville, VIC 3052, Australia.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.111

Reference46 articles.

1. Expression of Recombinant Human Methylmalonyl-CoA Mutase: In Primary mut Fibroblasts and Saccharomyces cerevisiae

2. Gene therapy for disorders affecting children, progress and potential

3. Long-term follow-up of 77 patients with isolated methylamalonic acidaemia

4. Rapid Diagnosis of Methylmalonic and Propionic Acidemias

5. Metabolic stroke in methylmalonic acidemia five years after liver transplantation

Cited by 15 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. Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors;Archives de Pédiatrie;2023-11

2. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia;Journal of Inherited Metabolic Disease;2023-08-07

3. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9;Molecular Therapy - Methods & Clinical Development;2022-12

4. Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia;Journal of Inherited Metabolic Disease;2022-07-21

5. Mitochondrial disease, mitophagy, and cellular distress in methylmalonic acidemia;Cellular and Molecular Life Sciences;2021-09-15

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