![]() ![]() Using a technology called RNA sequencing to look at how the mutation changed gene expression in these lines, the researchers then traced the changes to their impact on biological pathways. In the Cell Stem Cell study, Eggan and postdoctoral fellow Evangelos Kiskinis led an effort to make stem cell lines from two women with ALS who have SOD1 mutations in order to compare human biology and mouse biology. “And so,” he continued, “the key question that we really wanted to address was: Are clinical efforts failing because the mouse is taking us on a wild goose chase, or is it simply that people haven’t had the opportunity to pretest whether their ideas are true across lots of forms of ALS?” “The big problem in ALS is that there are more than 100 mutations in dozens of genes that all cause the disease, but almost all of the therapeutics that have gone forward in the clinic have done so for just one of those mutations, SOD1, which almost everyone studies in mice,” said Eggan, a professor in Harvard’s Department of Stem Cell and Regenerative Biology. While several potential treatments have looked promising in mice, all proved disappointing in the clinic. GALLANT STEM CELL SKINUsing neurons derived from stem cells made from ALS patient skin cells, the two research teams conducted clinical trials of the anti-epilepsy medication on neurons in laboratory dishes, finding that it reduced the cells’ hyperexcitability.ĪLS is a devastating and currently untreatable degradation of motor neurons, the long nerve cells that connect the spinal cord to the muscles of the body. Now Eggan and HSCI colleague Clifford Woolf have found that the many independent mutations that cause ALS may be linked by their ability to trigger abnormally high activity in motor neurons. The work, laid out in two related advance online publications in April by Cell Stem Cell and Cell Reports, is the long-term fruit of studies by Harvard Stem Cell Institute (HSCI) principal faculty member Kevin Eggan, who in a 2008 Science paper first raised the possibility of using ALS patient-derived stem cells to better understand the disease and identify therapeutic targets for new drugs. The investigators all caution that a great deal of work needs to be done to assure the safety and efficacy of the treatment in ALS patients before physicians should start offering it. GALLANT STEM CELL TRIALThe researchers are now collaborating with Massachusetts General Hospital (MGH) to design an initial clinical trial testing the safety of the treatment in ALS patients. ![]() Hirschhorn is the brother of Philadelphia Inquirer politics editor Dan Hirschhorn, who mourned the loss on Twitter.Harvard stem cell scientists have discovered that a recently approved medication for epilepsy might be a meaningful treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, a uniformly fatal neurodegenerative disorder. Hirschhorn had moved to Miami Beach from Los Angeles three years ago. Coast Guard and the Florida Fish & Wildlife Conservation Commission, Hirschhorn was struck by a 38-foot motor boat while he was riding his electric foil surfboard near Miami Beach, the Miami Herald reported. ![]() Philly bagel shop lands in Food & Wine's top 50 in United StatesĪccording to the U.S.GoPuff gets $1.5 billion in new funding amid COVID-19 pandemic's online shopping surge.Philly-based Entercom rebrands as Audacy, beefs up podcast presence. ![]() He pitched the company on ABC's "Shark Tank" in November 2019, scoring an investment from Lori Greiner and Anne Wojcicki, the co-founder and CEO of 23andMe. Hirschhorn also was the founder and CEO of Gallant, a stem-cell banking and therapeutics service for pets. The company raised more than $47 million and was later acquired by Seattle-based Rover in 2017. Hirschhorn, 42, who grew up in the Philadelphia area and attended Swarthmore College, is best-known for founding the pet-sitting company DogVacay. Tech entrepreneur Aaron Hirschhorn died after a boat struck him while surfing in Miami Beach on Sunday. ![]()
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |