Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference11 articles.
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2. Disorders of propionate and methylmalonate metabolism;Fenton,2001
3. Methylmalonic acidemia;Manoli,1993
4. Long-term outcome in methylmalonic acidurias is influenced by the underlying defect (mut0, mut-, cblA, cblB);Horster;Pediatr. Res.,2007
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2. Gene Therapy in Patients with the Crigler–Najjar Syndrome;New England Journal of Medicine;2023-08-17
3. Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia;Journal of Inherited Metabolic Disease;2023-08-07
4. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9;Molecular Therapy - Methods & Clinical Development;2022-12
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