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Novel Adeno-associated Viral (AAV) Vectors to Treat Hereditary Methylmalonic Acidemia (MMA) Caused by Methylmalonyl-coA Mutase (MMUT) Deficiency
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...
Published: 10/28/2024   |   Inventor(s): Randy Chandler, Charles Venditti
Keywords(s): AAV, Acidemia, Adeno-associated, CAUSED, DEFICIENCY, Hereditary, Methylmalonic, Methylmalonyl-CoA, MMA, Mmut, Mutase, Novel, TREAT, vectors, viral, VPXXXX, WIXXXX, WJXXXX, XEXXXX
Category(s): TherapeuticArea > Cardiology, Application > Therapeutics, TherapeuticArea > Infectious Disease, TherapeuticArea > Ophthalmology, TherapeuticArea > Dental, TherapeuticArea > Oncology, TherapeuticArea > Endocrinology, Application > Research Materials
LRRK2 Inhibitors: Novel Treatment for Intestinal Bowel Disorders
Use of Leucine Rich Repeat Kinase 2 (LRRK2) inhibitors for the treatment of Intestinal Bowel Disorders (IBD) is disclosed. IBD is a broad term that describes conditions with chronic or recurring immune response and inflammation of the gastrointestinal tract. Crohn's disease and ulcerative colitis, two common forms of idiopathic IBD, are chronic, relapsing...
Published: 10/28/2024   |   Inventor(s): Tetsuya Takagawa, Atsushi Kitani, Warren Strober
Keywords(s): COLITIS, Crohn's, Disease, IB3XXX, inhibitor, LRRK2, TREAT, Ulcerative, VPXXXX, WKXXXX, XEXXXX, YAXXXX, YBXXXX, YCXXXX
Category(s): Collaboration Sought > Licensing, Application > Therapeutics, TherapeuticArea > Endocrinology, TherapeuticArea > Infectious Disease, TherapeuticArea > Dental, TherapeuticArea > Ophthalmology, TherapeuticArea > Immunology, TherapeuticArea > Oncology, TherapeuticArea > Cardiology