Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy
Author:
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference38 articles.
1. Disorders of propionate and methylmalonate metabolism;Fenton,2001
2. The natural history of the inherited methylmalonic acidemias;Matsui;N Engl J Med,1983
3. Clinical outcome of long-term management of patients with vitamin B12-unresponsive methylmalonic acidemia;van der Meer;J Pediatr,1994
4. Neurological outcome of methylmalonic acidaemia;Nicolaides;Arch Dis Child,1998
5. Methylmalonic and propionic acidaemias: management and outcome;de Baulny;J Inherit Metab Dis,2005
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2. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia;Journal of Inherited Metabolic Disease;2023-08-07
3. Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia;Journal of Inherited Metabolic Disease;2023-06-06
4. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9;Molecular Therapy - Methods & Clinical Development;2022-12
5. Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia;PLOS ONE;2022-09-20
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