Protective genetic mutation offers new hope for understanding autism and brain development
Tel Aviv University researchers have made a discovery that could enhance our understanding of genetic mutations and their ...
New RAS Gene Discovery Unveils Hidden Pathway Driving Cancer Growth
These findings could pave the way for new treatment approaches for cancers driven by RAS mutations. Researchers at the ...
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Scientists develop worlds-first universal gene therapy for anemia
Interestingly, some DBA cases even result from direct GATA1 mutations. Although gene therapies targeting specific genetic ...
Genetic variation enhances cancer drug sensitivity in specific patient groups, study shows
By exploiting the genetic variation in cancer cells, an already approved cancer drug demonstrated enhanced effects against ...
The American Journal of Managed Care11h
FDA Approves Gene Therapy to Treat AADC Deficiency
The approval marks the first time gene therapy will be available to treat patients with aromatic I-amino acid decarboxylase ...
New tool allows researchers to study gene mutation directly within living human cells
Gene mutations have consequences both good and bad—from resistance to conditions like diabetes to susceptibility to certain ...
BioSpace14h
PTC Wins First FDA Nod for Direct-to-Brain Gene Therapy, Targets Ultrarare Disease
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene ...
Medpage Today on MSN22h
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...