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gene therapy
gene therapy
| 4 min read
by
Jennifer Tsang, PhD
Engineering bacteriophages to produce proteins in the gut
By using phages to deliver genes for therapeutic proteins, biologic drug delivery could become much easier.
News
| 3 min read
by
Alejandra Manjarrez, PhD
Batten disease hits the gut, but gene therapy may help
A gene therapy targeting organs beyond the brain could improve quality of life and possibly extend survival in children with the neurodegenerative disease.
News
| 5 min read
by
Dika Ojiakor, PhD
Tapping into tRNAs to treat rare diseases
Michelle Werner and her team at Alltrna are developing novel tRNA therapeutics to overwrite mutations shared by hundreds of rare genetic diseases.
News
| 6 min read
by
Maggie Chen
Finding new strategies to treat Pompe disease
Enzyme replacement therapy has been the mainstay of Pompe disease. New and emerging therapies may provide more options for patients.
News
| 3 min read
by
Jennifer Tsang, PhD
Toward an AAV gene therapy to treat muscular dystrophies
New dual AAV vector delivery overcomes challenges in delivering large genes.
News
| 3 min read
by
Jennifer Tsang, PhD
First brain-delivered AAV therapy approved by FDA
The gene therapy treats the rare genetic disease AADC deficiency by delivering a functional gene into the brain.
News
| 4 min read
by
Allison Whitten, PhD
and
Stephanie DeMarco, PhD
Clinical developments experts have their eye on in 2025
Industry leaders name the clinical trials and developments they’re most excited to see in the new year.
News
| 8 min read
by
Allison Whitten, PhD
Gene therapy could treat Alzheimer’s disease
New gene therapies in clinical trials seek to treat Alzheimer’s disease and heal the brain with a dose of helpful genetic material.
News
| 4 min read
by
Maggie Chen
CRISPR strategy could restore delayed-onset hearing loss
Previous genome editing techniques only work when delivered early in life. This approach could help preserve hearing that is lost later on.
News
| 4 min read
by
Samantha Borje
Delivering larger genes in gene therapy
A new method called “REVeRT” expands the capabilities of gene delivery platforms by transporting larger genes than previously possible.
Articles
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Volume 21 • Issue 1 • March 2025
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