No More Transplants? New Gene Therapy Targets Blood Stem Cells Directly

No More Transplants? New Gene Therapy Targets Blood Stem Cells Directly

Researchers have identified an early postnatal window that allows gene transfer to circulating blood stem cells, advancing the development of potential treatments for genetic disorders. A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has discovered a specific period shortly after birth when circulating blood stem cells can [...]

Lower Transfusion Thresholds May Be Safe for Children Post-Transplant

A UK study suggests lower red blood cell transfusion thresholds may be safe for children after stem cell transplant, showing high protocol adherence and no drop in quality of life.

UC San Diego Researchers Develop Gene Therapy Targeting Brain Cell Behavior in Alzheimer’s Treatment

Researchers at the University of California San Diego S […] The post UC San Diego Researchers Develop Gene Therapy Targeting Brain Cell Behavior in Alzheimer’s Treatment first appeared on GeneOnline News. The post UC San Diego Researchers Develop Gene Therapy Targeting Brain Cell Behavior in Alzheimer’s Treatment appeared first on GeneOnline News.

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 intrathecally quarterly, both showing similar safety and efficacy profiles.