UCSF News: Infant Gene Therapy is a Breakthrough for Artemis-SCID Patients

By Jess Berthold on December 22, 2022
Ten young children born without functioning immune systems and lacking the ability to fight infections are on track for healthier lives thanks to a new gene therapy treatment pioneered at UC San Francisco, reports a Dec. 22 study in the New England Journal of Medicine.

NYT Opinion: We Can Cure Disease by Editing a Person’s DNA. Why Aren’t We?

By Fyodor Urnov on December 09, 2022
The parents of a 2-year-old girl write that their daughter “could die within the next year” because a genetic mutation is causing her heart to fail. “Time is quickly running out for me,” writes a man in his mid-30s whose DNA harbors a genetic mistake certain to destroy his brain within a matter of...

UCSF News: Accelerating Transformational Research into Cell Transplantation for Patients with Type 1 Diabetes

By Kate Rix and Susan Godstone on July 22, 2022
A personal investigation into the lifelong implications of his type 1 diabetes (T1D) culminated in a $7 million gift from Mike Gordon, co-founder of Meritech Capital Partners, and his wife, Loren, to help UCSF surmount a key impediment to treating the disease. The funds will support world-class...

UCSF News: Sickle Cell Clinical Trial Aims to Cure Disease by Correcting Patient’s Mutated Gene

By Lorna Fernandes on December 01, 2021
UCSF Benioff Children’s Hospital Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease. The study, which plans to enroll its first patient later this year, will use CRISPR-Cas9 gene-editing technology on...

UCSF News: Living Therapeutics Initiative Will Accelerate Development and Delivery of Revolutionary Treatments

By Ariel Bleicher and Cyril Manning on June 10, 2021
UC San Francisco is launching a new initiative to propel the development of living therapeutics – a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease – and bring them quickly to patients. 

CIRM: Regulated, reliable, and reputable: Protect patients with uniform standards for stem cell treatments

By Geoffrey P. Lomax, Art Torres and Maria T. Millan on January 24, 2020
ABSTRACT - treatments are emerging amidst reports of patients being injured by unproven “stem cell” interventions. At this juncture, it is vital to be supporting the continued development of promising regenerative medicine products while protecting patients from the risks posed by unproven...

Gene Therapy For Inherited Blood Disorder Reduced Transfusions

By Rob Stein on April 18, 2018
Gene therapy is showing promise for treating one of the most common genetic disorders. Results of a study published Wednesday show that 15 of 22 patients with beta-thalassemia who got gene therapy were able to stop or sharply reduce the regular blood transfusions they had needed to alleviate their...

UCSF Benioff Children’s Hospitals in Oakland and San Francisco To Begin Cutting Edge Stem-Cell Trials at CIRM-Funded Alpha Stem Cell Clinics

By Melinda Krigel on December 11, 2017
California Institute of Regenerative Medicine Awards Nearly $8 Million to UCSF for the Clinics