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In vivo base editing extends lifespan of a humanized mouse model of prion disease.
Nat Med
January 2025
Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Prion disease is a fatal neurodegenerative disease caused by the misfolding of prion protein (PrP) encoded by the PRNP gene. While there is currently no cure for the disease, depleting PrP in the brain is an established strategy to prevent or stall templated misfolding of PrP. Here we developed in vivo cytosine and adenine base strategies delivered by adeno-associated viruses to permanently modify the PRNP locus to achieve PrP knockdown in the mouse brain.
View Article and Find Full Text PDFRobust genome editing activity and the applications of enhanced miniature CRISPR-Cas12f1.
Nat Commun
January 2025
Department of Physiology, Korea University College of Medicine, Seoul, Republic of Korea.
With recent advancements in gene editing technology using the CRISPR/Cas system, there is a demand for more effective gene editors. A key factor facilitating efficient gene editing is effective CRISPR delivery into cells, which is known to be associated with the size of the CRISPR system. Accordingly, compact CRISPR-Cas systems derived from various strains are discovered, among which Un1Cas12f1 is 2.
View Article and Find Full Text PDFCRISPR/Cas9-editing of PRNP in Alpine goats.
Vet Res
January 2025
UVSQ, INRAE, BREED, Université Paris-Saclay, 78350, Jouy-en-Josas, France.
Misfolding of the cellular PrP (PrP) protein causes prion disease, leading to neurodegenerative disorders in numerous mammalian species, including goats. A lack of PrP induces complete resistance to prion disease. The aim of this work was to engineer Alpine goats carrying knockout (KO) alleles of PRNP, the PrP-encoding gene, using CRISPR/Cas9-ribonucleoproteins and single-stranded donor oligonucleotides.
View Article and Find Full Text PDFEvidence Communication Rules for Policy (ECR-P) critical appraisal tool.
Syst Rev
January 2025
Statistical Laboratory, Department of Pure Mathematics and Mathematical Statistics, University of Cambridge, Cambridge, UK.
Background: Scientific papers increasingly put forward scientific-based policy recommendations (SPRs) as a means of closing the circle of science, policy and practice. Assessing the quality of such SPRs is crucial, especially within the context of a systematic review. Here, we present ECR-P (Evidence Communication Rules for Policy)-a critical appraisal tool that we have developed, which can be used in assessing not only the quality of SPRs but also the quality of their evidence base and how effectively these have both been communicated.
View Article and Find Full Text PDFEvaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.
bioRxiv
December 2024
Spotlight Therapeutics, Hayward, CA, USA.
Genetic medicines, including CRISPR/Cas technologies, extend tremendous promise for addressing unmet medical need in inherited retinal disorders and other indications; however, there remain challenges for the development of therapeutics. Herein, we evaluate genome editing by engineered Cas9 ribonucleoproteins (eRNP) in vivo via subretinal administration using mouse and pig animal models. Subretinal administration of adenine base editor and double strand break-inducing Cas9 nuclease eRNPs mediate genome editing in both species.
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