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1. CRISPR Therapeutics
Perhaps the most exciting recent study in diabetes has been the use of clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9, the gene-editing tool, to reverse diabetes. One of the primary ways diabetes develops is a shortage of insulin-producing beta cells in the pancreas. A 2016 study sponsored by the National Institutes of Health showed that transplanting pancreatic cells can come close to curing type 1 diabetes. That procedure comes with some drawbacks. It requires a hard-to-find cadaver donor, and the recipient must take immunosuppressant drugs to prevent the body from rejecting the transplant.
To get around those constraints, ViaCyte and CRISPR joined forces in 2018, using CRISPR, to engineer stem cells to be immune-evasive, insulin-producing pancreatic cells. Preliminary results have shown that these cells do indeed mature into normal human pancreatic tissue. That could serve as a functional cure for many
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An experimental cell therapy for Type 1 diabetes has hit a significant milestone, as its developer, Vertex, announced Wednesday the start of initial testing in humans.
Vertex got its hands on the therapy, known as VX-880, through the $1 billion acquisition of Semma Therapeutics. Semma was co-founded by Douglas Melton, whose lab at Harvard University gave rise to VX-880.
An early-stage study of the therapy was posted March 8, and expects to enroll about 17 patients who have Type 1 diabetes along with severely low blood sugar and impaired ability to sense it. The study will look primarily at how safe and tolerable the treatment is, as well as the proportion of participants who aren t having severe hypoglycemic events afterwards. It s estimated that data collection for the primary endpoint will wrap up in early 2024.
Douglas-meltonBastiano-sannaHarvard-universityDrug-administrationDive-briefTrack-designationSan-diego-based-viacyteBiotechடக்ளஸ்-மெல்டன்ஹார்வர்ட்-பல்கலைக்கழகம்டைவ்-சுருக்கமானடிராக்-பதவிUMass Medical School joins JDRF Center of Excellence in New England to advance type 1 diabetes research
Center will focus on accelerating gene editing approaches for beta cell replacement therapy By Colleen Locke and Bryan Goodchild
UMass Medical School Communications February 23, 2021
Five UMass Medical School scientists from the UMass Diabetes Center of Excellence are part of the JDRF Center of Excellence in New England that launched Tuesday, Feb. 23. The goal of the cross-institutional collaboration is to create a cell replacement therapy that could treat and potentially cure type 1 diabetes.
Funded by JDRF and led by Harvard Stem Cell Institute co-director Douglas Melton, PhD, and JDRF scientific staff leader Esther Latres, PhD, assistant vice president of research, the new center comprises leading Massachusetts-based experts, including Michael Brehm, PhD, and Dale L. Greiner, PhD, of UMass Medical School, and Stephan Kissler, PhD, o
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