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The repurposing of the CRISPR/Cas bacterial defense system against bacteriophages as simple and flexible molecular tools has revolutionized the field of gene editing. These tools are now widely used in basic research and clinical trials involving human somatic cells. However, a global moratorium on all clinical uses of human germline editing has been proposed because the technology still lacks the required efficacy and safety. Here we focus on the approaches developed since 2013 to decrease the frequency of unwanted mutations (the off-targets) during CRISPR-based gene editing.
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http://dx.doi.org/10.3389/fcell.2021.718466 | DOI Listing |
Clin Appl Thromb Hemost
September 2025
Pediatric Hematology Laboratory, Division of Hematology/Oncology, Department of Pediatrics, The Seventh Affiliated Hospital of Sun Yat-Sen University, Shenzhen, Guangdong, China.
Hemophilia, an X-linked monogenic disorder, arises from mutations in the or genes, which encode clotting factor VIII (FVIII) or clotting factor IX (FIX), respectively. As a prominent hereditary coagulation disorder, hemophilia is clinically manifested by spontaneous hemorrhagic episodes. Severe cases may progress to complications such as stroke and arthropathy, significantly compromising patients' quality of life.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
September 2025
Office of Gene Therapy, Office of Therapeutic Products, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD 20993, USA.
genome editing with CRISPR-Cas9 systems is generating worldwide attention and enthusiasm for the possible treatment of genetic disorders. However, the consequences of potential immunogenicity of the bacterial Cas9 protein and the AAV capsid have been the subject of considerable debate. Here, we model the antigen presentation in cells after gene editing by transduction of a human cell line with an AAV2 vector that delivers the Cas9 transgene.
View Article and Find Full Text PDFClin Kidney J
September 2025
Department of Translational Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy.
Genome editing technologies, particularly clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9, have transformed biomedical research by enabling precise genetic modifications. Due to its efficiency, cost-effectiveness and versatility, CRISPR has been widely applied across various stages of research, from fundamental biological investigations in preclinical models to potential therapeutic interventions. In nephrology, CRISPR represents a groundbreaking tool for elucidating the molecular mechanisms underlying kidney diseases and developing innovative therapeutic approaches.
View Article and Find Full Text PDFJ Appl Stat
February 2025
Department of Mathematics and State Key Laboratory of Novel Software Technology, Nanjing University, Nanjing, People's Republic of China.
We conduct gene mutation rate estimations via developing mutual information and Ewens sampling based convolutional neural network (CNN) and machine learning algorithms. More precisely, we develop a systematic methodology through constructing a CNN. Meanwhile, we develop two machine learning algorithms to study protein production with target gene sequences and protein structures.
View Article and Find Full Text PDFNat Aging
September 2025
Aging Biomarker Consortium (ABC), Beijing, China.
The global surge in the population of people 60 years and older, including that in China, challenges healthcare systems with rising age-related diseases. To address this demographic change, the Aging Biomarker Consortium (ABC) has launched the X-Age Project to develop a comprehensive aging evaluation system tailored to the Chinese population. Our goal is to identify robust biomarkers and construct composite aging clocks that capture biological age, defined as an individual's physiological and molecular state, across diverse Chinese cohorts.
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