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Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency
Case ID:
TAB-3619
Web Published:
12/6/2022
Description:
Deficiency of the enzyme in methylmalonyl-CoA mutase (MMUT) results is a life-threatening disease, methylmalonic acidemia (MMA), that carries high rates of morbidity and mortality. NHGRI scientists have developed novel AAV vectors that combine the proprietary codon-optimized synMMUT alleles with either a liver-specific promoter from the human alpha-1 antitrypsin (hAA T) locus to produce a vector that directs MMUT protein expression in a liver-specific fashion or the human elongation factor 1a (EF1 alpha) promoter to produce a vector that expresses the MMUT protein at moderate levels in a global fashion, including the liver. These AAV vectors have high potency in vivo, and therefore represent a class of new gene therapies that might be given to patients with MMA. The AAV constructs developed and enabled as serotype 8 or 9 vectors could be immediately translated to the clinic.
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Direct Link:
https://nih.technologypublisher.com/tech/Novel_Adeno-associated_Viral_(AAV)_V ectors_to_Treat_Hereditary_Methylmalonic_Acidemia_(MMA)_Caused_by_Methylmalo nyl-coA_Mutase_(MMUT)_Deficiency
Category(s):
Cardiology
Therapeutics
Infectious Disease
Ophthalmology
Dental
Oncology
Endocrinology
Research Materials
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For Information, Contact:
Eggerton Campbell
Licensing And Patenting Manager
NIH Technology Transfer
301-402-1648
eggerton.campbell@nih.gov
Inventors:
Randy Chandler
Charles Venditti
Keywords:
AAV
Acidemia
Adeno-associated
CAUSED
DEFICIENCY
Hereditary
Methylmalonic
Methylmalonyl-CoA
MMA
Mmut
Mutase
Novel
TREAT
vectors
viral
VPXXXX
WIXXXX
WJXXXX
XEXXXX
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