The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility. The new funding will help the research…
St. Jude Children’s Research Hospital scientists identified a promising combination approach to treat a rare, but catastrophic pediatric brain cancer called atypical teratoid rhabdoid tumor (ATRT). Reactivating and maintaining p53, a protein responsible for tumor suppression, using the drugs idasantulin and selinexor, respectively, was well-tolerated, reduced tumor burden and increased survival in a laboratory model….
Texas Children’s is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency. Aromatic l-amino acid decarboxylase (AADC) deficiency is an extremely rare, inherited neurological disorder that prevents the brain from producing dopamine and serotonin – essential chemicals for controlling movement, mood and basic…