New discovery enables gene therapy for muscular dystrophies, …
22 hours ago · StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models. Gene therapy can effectively treat various ...
The First Crispr Treatment Is Making Its Way to Patients
1 day ago · Nov 15, 2024 5:30 AM. The First Crispr Treatment Is Making Its Way to Patients. It’s been a year since the gene-editing treatment Casgevy was approved for sickle cell disease and a related blood ...
New Discovery Enables Gene Therapy for Muscular Dystrophies, …
2 days ago · People with limb girdle muscular dystrophy experience weakness and wasting in the shoulder, hip, and thigh muscles, and often have difficulty standing, moving, and doing everyday tasks. “Gene therapy is a powerful tool for delivering a healthy gene copy back to a patient’s cells to correct genetic diseases, but the vectors used to deliver ...
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
1 day ago · Opens in a new tab or window. Share on X. Opens in a new tab or window. ... The treatment is the first-ever gene therapy approved in the U.S. directly administered to the brain, ...
FDA approves first gene-editing treatment for human illness
Dec 8, 2023 · The Food and Drug Administration approved two genetic treatments for sickle cell disease, including one that uses gene-editing. The approvals offer hope for patients and signal a new...
Gene Therapy News - ScienceDaily
3 days ago · Read all about gene therapy including current medical research on switching therapeutic genes on and off, light-activated gene therapy and gene silencing.
FDA Approves First Gene Therapies to Treat Patients with Sickle …
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first...
FDA approves cure for sickle cell disease, the first treatment to use ...
Dec 8, 2023 · The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a...
Next-generation CRISPR-based gene-editing therapies tested in
Aug 5, 2024 · With the first CRISPR–Cas9 gene therapy now approved, scientists are turning to newer editing technologies to produce safer, faster and better treatments for genetic diseases.
A new age of precision gene therapy - The Lancet
Nov 23, 2023 · Gene therapy has become a clinical reality as market-approved advanced therapy medicinal products for the treatment of distinct monogenetic diseases and B-cell malignancies.