Drexel University

Breakthrough on Gene Therapy for Hereditary Spastic Paraplegia

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

Gene therapy for hereditary spastic paraplegia hits proof-of-principle milestone

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College ...
Time

A Huntington’s Disease Treatment Is Closer Than Ever

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

Exa-cel gene therapy may off effective cure for beta-thalassemia and sickle cell disease in children younger than 12

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...
The New York Times

New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases

Instead of requiring personalized gene edits for each patient, the new approach could create a standardized method to use for many diseases. By Pam Belluck and Carl Zimmer Gene-editing therapies offer ...

Teens may have come up with a new way to detect, treat Lyme disease using CRISPR gene editing

To compete at iGEM, a sort of science Olympics, teens at a Georgia high school set their sights on finding a better way to detect and treat Lyme disease. Their approach uses CRISPR gene editing.
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
The American Journal of Managed Care

Splicing Modifiers and Gene Therapy for SMA

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...
News-Medical.Net on MSN

Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia ...
MarketWatch

SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines

The MarketWatch News Department was not involved in the creation of this content. -- Financing co-led by new investors EQT Life Sciences and Sanofi Ventures, with participation from Roche Venture Fund ...
Business Wire

Cure Rare Disease Secures FDA Orphan Drug Designation for Investigational Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type R9 (LGMD2i/R9)

WOODBRIDGE, Conn.--(BUSINESS WIRE)--Cure Rare Disease (CRD), a 501(c)(3) nonprofit biotechnology company developing genetic therapies for ultra-rare neuromuscular diseases, today announced that the ...
Hosted on MSN

Activated immune cells reveal hidden drivers of autoimmune diseases

Subscribe to our newsletter for the latest sci-tech news updates. Published in Nature Communications, scientists from the Wellcome Sanger Institute reveal insights into how our genes influence the ...