Scientists at The Hospital for Sick Children (SickKids) have identified a key gene that could lead to next-generation treatments for medulloblastoma, the most common malignant brain tumor in children.

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the use of personalized antisense oligonucleotides (ASOs). This innovative ...

Recurrent respiratory papillomatosis (RRP) is a disease of the upper airway presenting with exophytic wart-like lesions. These lesions are considered benign with less than a 1–5% chance of malignant ...

Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

Intraperitoneal delivery of adenoviral gene therapy in vivo is safe and nontoxic. The use of new diluents, already used in peritoneal renal dialysis, will improve drug delivery, reduce therapeutic ...

UC San Diego Health is one of a few health care systems on the West Coast to offer a new gene therapy for hemophilia B, a bleeding disorder that prevents blood from clotting properly. The FDA-approved ...

Anger management therapy can include a host of different therapies such as cognitive behavioral therapy or psychodynamic therapy. Your therapist or psychologist will tailor any chosen therapy to ...

Gene-editing kicked off 2024 with a bang in the medical sector but closed the year with a whimper. It started with the FDA approval for the first CRSPR-based gene therapy treatment for sickle cell ...

Some genes are beneficial and enhance longevity. For example, the gene that helps a person metabolize cholesterol would reduce a person's risk of heart disease. Some gene mutations are inherited.

To explore gene therapy approaches relevant to the inhibition of NF-κB, we used cDNA encoding the 'super-repressor' form of IκBα (ref. 19) cloned into an adenovirus 5 vector to produce ...

Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...