Therapeutic targeting of RNA for neurological and neuromuscular disease
JL Bubenik, MM Scotti, MS Swanson - Genes & Development, 2024 - genesdev.cshlp.org
Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo
mutations have devasting personal, familial, and societal impacts. As the initial product of …
mutations have devasting personal, familial, and societal impacts. As the initial product of …
Leveraging CRISPR gene editing technology to optimize the efficacy, safety and accessibility of CAR T-cell therapy
T Lei, Y Wang, Y Zhang, Y Yang, J Cao, J Huang… - Leukemia, 2024 - nature.com
Abstract Chimeric Antigen Receptor (CAR)-T-cell therapy has revolutionized cancer immune
therapy. However, challenges remain including increasing efficacy, reducing adverse events …
therapy. However, challenges remain including increasing efficacy, reducing adverse events …
CRISPR–Cas13: Pioneering RNA editing for nucleic acid therapeutics
G Zhu, X Zhou, M Wen, J Qiao, G Li, Y Yao - BioDesign Research, 2024 - spj.science.org
The CRISPR–Cas13 system has emerged as a revolutionary tool for RNA editing, offering
new opportunities for the development of nucleic acid therapeutics. Unlike DNA-targeting …
new opportunities for the development of nucleic acid therapeutics. Unlike DNA-targeting …
Precision Transcriptome Editing
C Chen, LS Qi - ACS Synthetic Biology, 2024 - ACS Publications
Manipulating RNA species in mammalian cells has emerged as an important strategy for
precise gene expression control. Here we review recent advances in precision …
precise gene expression control. Here we review recent advances in precision …
Chemical engineering of CRISPR–Cas systems for therapeutic application
Clustered regularly interspaced short palindromic repeats (CRISPR) technology has
transformed molecular biology and the future of gene-targeted therapeutics. CRISPR …
transformed molecular biology and the future of gene-targeted therapeutics. CRISPR …
LncRNAs, RNA Therapeutics, and Emerging Technologies in Liver Pathobiology
AA Anwar, N Jalan-Sakrikar… - Seminars in Liver …, 2024 - thieme-connect.com
The field of ribonucleic acid (RNA) biology has revealed an array of noncoding RNA
species, particularly long noncoding RNAs (lncRNAs), which play crucial roles in liver …
species, particularly long noncoding RNAs (lncRNAs), which play crucial roles in liver …
A Manual for Genome and Transcriptome Engineering
Y Doctor, M Sanghvi, P Mali - IEEE Reviews in Biomedical …, 2024 - ieeexplore.ieee.org
Genome and transcriptome engineering have emerged as powerful tools in modern
biotechnology, driving advancements in precision medicine and novel therapeutics. In this …
biotechnology, driving advancements in precision medicine and novel therapeutics. In this …
Recent Therapeutic Gene Editing Applications to Genetic Disorders
E Deneault - Current Issues in Molecular Biology, 2024 - mdpi.com
Recent years have witnessed unprecedented progress in therapeutic gene editing,
revolutionizing the approach to treating genetic disorders. In this comprehensive review, we …
revolutionizing the approach to treating genetic disorders. In this comprehensive review, we …