News

From birth to gene-edited in 6 months: Custom therapy breaks speed limits
The researchers described the unprecedented speed of the oversight steps as being "through alternative procedures." In month ...
Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions
A baby born with a rare and dangerous genetic disease is thriving after getting an experimental gene editing treatment made ...
Children's Hospital of Philadelphia Researchers Develop Promising Tools to Enhance Gene Therapy Delivery for Hard-to-Treat Diseases
Adeno-associated viral (AAV) vectors are promising tools that can transport modified genetic material into the nuclei of cells in target tissues impacted by challenging diseases, offering the ...
Discover Magazine on MSN3d
CRISPR Fulfills Its Promise with First-Ever Personalized Gene-Editing Therapy
When CRISPR was first introduced as a gene-editing tool in 2012, the world was in awe of all the possibilities it held - ...
Child treated with gene editing therapy for the first time by a team of Philadelphia doctors
A team of doctors at the Children's Hospital of Philadelphia and Penn Medicine treated a baby with a gene editing technology ...
CHOP and Penn treated an infant with a rare disease by editing his genes
A Delaware County infant with a rare metabolic disease, CPS1 deficiency, was treated at CHOP with a custom CRISPR therapy.
AAN releases Evidence in Focus Article on new gene therapy for Duchenne muscular dystrophy
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of ...
News Medical on MSN4d
New gene editor evoCAST enables precise insertion of complete genes
Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia ...
Medscape8d
Hemophilia B: Post-Beqvez, What’s Next for Gene Therapy?
In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later ...