By exploiting a feature of the immune system, researchers open the door for stem cell transplants to repair the brain. In experiments in mice, Johns Hopkins Medicine researchers say they have developed a way to successfully transplant certain protective brain cells without the need for lifelong anti-rejection drugs. A report on the research, published Sept. 16 in
At the Association for Glycogen Storage Disease’s 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children’s presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease (GSD). The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously
In the body’s cells, some proteins are of vital importance as to which genes are active or turned off. Now, researchers from the University of Copenhagen and the Memorial Sloan Kettering Cancer Center have discovered which proteins are necessary in order to maintain the proper genetic regulation. All of the more than 200 different cell
Long-Term Follow-up Data Show That the 88% of Patients Treated in the Starbeam Study (ALD-102) Were Free of Major Functional Disabilities (MFDs) at Two Years, and Continued to Remain MFD-Free at up to Five Years of Follow-up bluebird bio, Inc. (Nasdaq: BLUE) today announced updated results from the clinical development program for its investigational Lenti-D™ gene
Source The Niche Knoepfler Lab Stem Cell Blog This afternoon Dr. Knoepfler attended and gave public comment at the Medical Board of California stem cell task force meeting on unproven stem cell clinics. There were about 40 people attending. In part this meeting is a consequence of the national governing organization of state medical boards, the Federation of
Source Cell Stem Cell Human pluripotent stem cells (hPSCs) are known to harbor chromosomal aberrations, affecting their tumorigenic potential. We established a strategy to identify cancer-related point mutations in hPSCs, detecting recurrent mutations in over 20 genes, alongside those previously detected in p53. Importantly, naive hPSCs harbor, on average, four times more mutations than their
New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who
Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus
In new research looking at pivotal trials for new cancer drugs approved by the European Medicines Agency between 2014 and 2016, researchers found only a quarter of trials use overall survival as a primary endpoint. Trials for cancer drugs undertaken in Europe in the mid-2010s were flawed to such a degree as to “raise questions”
In a small safety and feasibility clinical trial, Mayo Clinic researchers have demonstrated for the first time that senescent cells can be removed from the body using drugs termed “senolytics”. The result was verified not only in analysis of blood but also in changes in skin and fat tissue senescent cell abundance. This trial —
