Gene therapy, gene editing and perspectives on future use in … – PMLiVE

Gene therapy, gene editing and perspectives on future use in … – PMLiVE
Gene therapy, gene editing and perspectives on future use in ...  PMLiVE ... read more
Source: Google NewsPublished on 2023-05-26

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  • The road ahead: 2022 January 20, 2022 # # # # The first post at the start of each year on the SoPD website tries to provide an overview of where things are in the search for ‘disease modifying’ therapies for Parkinson’s.  It is an exercise in managing expectations as well as discussing what research events are scheduled for the next year so that we can keep…
  • The road ahead: 2021 January 25, 2021 # # # # At the start of each year, it is a useful practise to layout what is planned over the next 12 months. The events that are scheduled for the year to come, so that we can keep an eye out for them. Obviously, where 2021 will end actually is unpredictable, but an outline of what is scheduled…
  • Bayer doubles down on Parkinson’s? December 10, 2020 # # # # News today of two biotech companies merging did not cause much of a ripple in the media, but the wider implications of the move are rather significant for Parkinson’s. Today it was announced that Brain Neurotherapy Bio (BNB) is going to merge with Asklepios Biopharmaceutical (aka AskBio). BNB are currently clinically testing a GDNF gene therapy…
  • Prevail lands on a Lilly pad December 15, 2020 # # # # 2020 has been a dreadful year for most of the world – burdened by the outbreak and consequences of COVID-19. Despite this, there has been a steady stream of biotech acquisitions related to Parkinson’s which have helped to keep morale high in the PD research community. In October alone, we saw the Portuguese pharmaceutical company Bial…
  • Highly efficient generation of isogenic pluripotent stem cell models using prime editing September 7, 2022 Elife. 2022 Sep 7;11:e79208. doi: 10.7554/eLife.79208. Online ahead of print.ABSTRACTThe recent development of prime editing (PE) genome engineering technologies has the potential to significantly simplify the generation of human pluripotent stem cell (hPSC)-based disease models. PE is a multi-component editing system that uses a Cas9-nickase fused to a reverse transcriptase (nCas9-RT) and an extended PE guide RNA (pegRNA). Once reverse…
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  • Highly efficient generation of isogenic pluripotent stem cell models using prime editing September 7, 2022 Elife. 2022 Sep 7;11:e79208. doi: 10.7554/eLife.79208. Online ahead of print.ABSTRACTThe recent development of prime editing (PE) genome engineering technologies has the potential to significantly simplify the generation of human pluripotent stem cell (hPSC)-based disease models. PE is a multi-component editing system that uses a Cas9-nickase fused to a reverse transcriptase (nCas9-RT) and an extended PE guide RNA (pegRNA). Once reverse…
  • Genetic control of RNA editing in neurodegenerative disease January 22, 2023 Brief Bioinform. 2023 Jan 21:bbad007. doi: 10.1093/bib/bbad007. Online ahead of print.ABSTRACTA-to-I RNA editing diversifies human transcriptome to confer its functional effects on the downstream genes or regulations, potentially involving in neurodegenerative pathogenesis. Its variabilities are attributed to multiple regulators, including the key factor of genetic variants. To comprehensively investigate the potentials of neurodegenerative disease-susceptibility variants from the view of A-to-I…
  • Genetic control of RNA editing in neurodegenerative disease January 22, 2023 Brief Bioinform. 2023 Jan 21:bbad007. doi: 10.1093/bib/bbad007. Online ahead of print.ABSTRACTA-to-I RNA editing diversifies human transcriptome to confer its functional effects on the downstream genes or regulations, potentially involving in neurodegenerative pathogenesis. Its variabilities are attributed to multiple regulators, including the key factor of genetic variants. To comprehensively investigate the potentials of neurodegenerative disease-susceptibility variants from the view of A-to-I…
  • Genetic control of RNA editing in neurodegenerative disease January 22, 2023 Brief Bioinform. 2023 Jan 21:bbad007. doi: 10.1093/bib/bbad007. Online ahead of print.ABSTRACTA-to-I RNA editing diversifies human transcriptome to confer its functional effects on the downstream genes or regulations, potentially involving in neurodegenerative pathogenesis. Its variabilities are attributed to multiple regulators, including the key factor of genetic variants. To comprehensively investigate the potentials of neurodegenerative disease-susceptibility variants from the view of A-to-I…
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  • The Potential Regulation of A-to-I RNA Editing on Genes in Parkinson's Disease April 28, 2023 Genes (Basel). 2023 Apr 15;14(4):919. doi: 10.3390/genes14040919.ABSTRACTParkinson's disease (PD) is characterized by dopaminergic neurodegeneration and an abnormal accumulation of α-synuclein aggregates. A number of genetic factors have been shown to increase the risk of PD. Exploring the underlying molecular mechanisms that mediate PD's transcriptomic diversity can help us understand neurodegenerative pathogenesis. In this study, we identified 9897 A-to-I RNA editing…
  • Monthly research review: February 2020 February 29, 2020   At the end of each month the SoPD writes a post which provides an overview of some of the major pieces of Parkinson’s-related research that were made available during February 2020. The post is divided into seven parts based on the type of research: Basic biology Disease mechanism Clinical research New clinical trials Clinical trial news Other news Review…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • Transient inhibition of p53 enhances prime editing and cytosine base-editing efficiencies in human pluripotent stem cells October 27, 2022 Nat Commun. 2022 Oct 27;13(1):6354. doi: 10.1038/s41467-022-34045-7.ABSTRACTPrecise gene editing in human pluripotent stem cells (hPSCs) holds great promise for studying and potentially treating human diseases. Both prime editing and base editing avoid introducing double strand breaks, but low editing efficiencies make those techniques still an arduous process in hPSCs. Here we report that co-delivering of p53DD, a dominant negative fragment…
  • The road ahead: 2020 January 5, 2020 Here at the SoPD, we are primarily interested in disease modification for Parkinson’s. While there is a great deal of interesting research exploring the causes of the condition, novel symptomatic therapies, and other aspects of Parkinson’s, my focus is generally on the science seeking to slow, stop or reverse the condition. At the start of each year, it is a…
  • Lilly scores gene therapy programme in $1bn Prevail Therapeutics acquisition deal - PMLiVE December 17, 2020 Lilly scores gene therapy programme in $1bn Prevail Therapeutics acquisition deal  PMLiVE