Title:Precision Genome Editing Techniques in Gene Therapy: Current State
and Future Prospects
Volume: 24
Issue: 5
关键词:
精确基因组编辑,基因治疗,CRISPR-Cas9,遗传疾病,治疗干预,碱基编辑,引物编辑。
摘要: Precision genome editing is a rapidly evolving field in gene therapy, allowing for the
precise modification of genetic material. The CRISPR and Cas systems, particularly the CRISPRCas9
system, have revolutionized genetic research and therapeutic development by enabling precise
changes like single-nucleotide substitutions, insertions, and deletions. This technology has the
potential to correct disease-causing mutations at their source, allowing for the treatment of various
genetic diseases. Programmable nucleases like CRISPR-Cas9, transcription activator-like effector
nucleases (TALENs), and zinc finger nucleases (ZFNs) can be used to restore normal gene function,
paving the way for novel therapeutic interventions. However, challenges, such as off-target
effects, unintended modifications, and ethical concerns surrounding germline editing, require careful
consideration and mitigation strategies. Researchers are exploring innovative solutions, such as
enhanced nucleases, refined delivery methods, and improved bioinformatics tools for predicting
and minimizing off-target effects. The prospects of precision genome editing in gene therapy are
promising, with continued research and innovation expected to refine existing techniques and uncover
new therapeutic applications.