Gene Therapy Sees Children With Rare Genetic Condition Walk And Talk For First Time Aromatic l-amino acid decarboxylase (AADC) deficiency is a genetic disorder of the nervous system. Only about 120 cases have ever been reported, and around half of these have been in people of Asian ancestry. The condition is caused by mutations in the DDC gene, which
One of the dreams of cancer therapy is to use the power of the body to heal itself. This dream, long in the making, is becoming a reality thanks to deep and fundamental understandings of the immune system, the primary means by which we protect ourselves from external and internal threats. The immune system recognizes
Groundbreaking trial aims to reverse the paralysis and other abnormal functions of spina bifida before birth Three babies have been born after receiving the world’s first spina bifida treatment combining surgery with stem cells. This was made possible by a landmark clinical trial at UC Davis Health. The one-of-a-kind treatment, delivered while a fetus is
CARTITUDE-1 is a phase 1b–2 study evaluating ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor T cell therapy with two B-cell maturation antigen–targeting single-domain antibodies, in patients with relapsed or refractory multiple myeloma. Primary efficacy outcomes have previously been reported. In this paper are reported health-related quality of life (HRQOL) secondary outcomes evaluated using patient-reported outcomes.
The prognosis for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia remains poor. UCART19, an allogeneic genome-edited anti-CD19 chimeric antigen receptor (CAR) T-cell product derived from healthy donors and available for immediate clinical use, offers a potential therapeutic option for such patients. The CALM trial is a first-in-human study evaluating the safety and antileukaemic
Patients’ low-light sensitivity improved by factors of thousands in a clinical trial Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene therapy, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. The patients
A Yale Medicine orthopaedic surgeon has developed an innovative procedure that uses a patient’s own stem cells to treat a condition that can cause painful hip fractures. The condition, called avascular necrosis, is when the flow of blood to the bones is cut off or reduced. It can be caused by a trauma, such as a
In this short documentary video from the New England Journal of Medicine, patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy. Patients share their own stories of interactions with the health care system and explore
In preliminary results from a Phase I study of CLBR001 + SWI019 for patients with B cell malignancies: 7 of 9 patients responded and 6 of 9 had a complete response (78% ORR, 67% CR) CLBR001 cells engrafted at higher levels than approved CAR-T cell products without causing an increase in the incidence of CRS
Improvements in technology and delivery techniques could revolutionize treatment of genetic disorders of vision In a new paper, University of California, Irvine researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases (IRDs). The study, titled, “Precision genome editing in the eye,” was published this week
