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Development and optimization of base editors and its application in crops.
Biochem Biophys Res Commun
December 2024
College of Plant Protection, Shandong Agricultural University, Tai'an, 271018, China. Electronic address:
Article Synopsis
- Genome editing technologies, particularly base editing, show great promise for improving crops by enabling targeted changes at the genetic level, especially through single nucleotide variations known as SNPs.
- Base editing allows for precise modifications of DNA without causing double-strand breaks, making it a safer alternative to traditional CRISPR/Cas9 methods.
- The review highlights advancements in different base editing systems, discusses their applications in agriculture, addresses current limitations, and offers insights into their future potential.
To BE or to PE: Prime editors provide more choices for epitope-editing-based immunotherapy.
Cell Stem Cell
November 2024
Shanghai Frontiers Science Center of Genome Editing and Cell Therapy, Shanghai Key Laboratory of Regulatory Biology, Institute of Biomedical Sciences and School of Life Sciences, East China Normal University, Shanghai, China. Electronic address:
Epitope editing is a promising strategy for protecting hematopoietic cells from eradication by immunotherapies. Recently, in Cell Stem Cell, Ji et al. applied both base editing (BE) and prime editing (PE) to alter the epitope of CD123 in hematopoietic stem cells for CAR-T therapy against acute myeloid leukemia.
View Article and Find Full Text PDFCell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy.
Hum Gene Ther
October 2024
Oswaldo Cruz Foundation (FIOCRUZ), National Institute of Women, Children and Adolescents' Health Fernandes Figueira (IFF), Rio de Janeiro, Brazil.
The advent of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated nuclease 9 (Cas9) technology has revolutionized the field of genetic engineering, offering unprecedented potential for the targeted manipulation of DNA sequences. Advances in the mechanism of action of the CRISPR-Cas9 system allowed potential applicability for the treatment of genetic diseases. CRISPR-Cas9's mechanism of action involves the use of an RNA guide molecule to target-specific DNA sequences and the Cas9 enzyme to induce precise DNA cleavage.
View Article and Find Full Text PDFPE6c greatly enhances prime editing in transgenic rice plants.
J Integr Plant Biol
September 2024
State Key Laboratory of Plant Environmental Resilience, College of Biological Sciences, China Agricultural University, Beijing, 100193, China.
Prime editing is a versatile CRISPR/Cas-based precise genome-editing technique for crop breeding. Four new types of prime editors (PEs) named PE6a-d were recently generated using evolved and engineered reverse transcriptase (RT) variants from three different sources. In this study, we tested the editing efficiencies of four PE6 variants and two additional PE6 constructs with double-RT modules in transgenic rice (Oryza sativa) plants.
View Article and Find Full Text PDFiSCORE-PD: an isogenic stem cell collection to research Parkinson's Disease.
bioRxiv
February 2024
Dominick P. Purpura Department of Neuroscience, Albert Einstein College of Medicine, Rose F. Kennedy Center, Albert Einstein College of Medicine, 1410 Pelham Parkway South, Bronx, NY 10461, USA.
Parkinson's disease (PD) is a neurodegenerative disorder caused by complex genetic and environmental factors. Genome-edited human pluripotent stem cells (hPSCs) offer the uniique potential to advance our understanding of PD etiology by providing disease-relevant cell-types carrying patient mutations along with isogenic control cells. To facilitate this experimental approach, we generated a collection of 55 cell lines genetically engineered to harbor mutations in genes associated with monogenic PD ( A53T, A30P, Ex3del, Q129X, Ex1-5del, G2019S, FS, R498X/FS, c.
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