WebMar 9, 2024 · On 28 February, Intellia Therapeutics in Cambridge, Massachusetts, announced that its experimental CRISPR–Cas9 treatment for a rare condition called … WebAug 18, 2024 · To validate the results, they ran a pooled CRISPR screen on 42 of those high-confidence variants in multiple cell types. ... Functional regulatory variants implicate distinct transcriptional networks in dementia. INTRODUCTION. The widespread adoption of genome-wide association studies (GWASs) has revolutionized the detection of genetic …
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WebApr 11, 2024 · CRISPR reveals a combination strategy The researchers found that loss of the SPOP gene causes significant BET inhibitor resistance, which they confirmed in cell lines and xenograft mouse models. WebMay 24, 2024 · By integrating CRISPR-based functional genomics and stem cell technology, researchers based at the University of California, San Francisco (UCSF), have … click on telefonica
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WebNov 23, 2024 · CRISPR-Cas9 is a promising tool for modeling and treating genetic diseases. This system has been used in cell lines, induced pluripotent stem cells (iPSCs) derived from patients, and animal models. Here, we mainly focus on its … WebMar 29, 2024 · CURRENT CRISPR CLINICAL TRIALS. In the first use of an ex vivo CRISPR-based therapy to treat a genetic disease, researchers treated an individual with … WebHexanucleotide-repeat expansions in the C9ORF72 gene are the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (c9ALS/FTD). The nucleotide-repeat expansions are translated into dipeptide-repeat (DPR) proteins, which are aggregation prone and may contribute to neurodegene … click on text