Review Article
Open Access
Recent Advances in Gene Editing and Gene Therapy: Transforming Precision Medicine
Feng Jhang *, Mikako Wada , Davis John, Jennifer Doud
Broad Institute of MIT and Harvard, Merkin Building, 415 Main St, Cambridge, MA 02142, United States
Feng Jhang, et al. Recent Advances in Gene Editing and Gene Therapy:Transforming Precision Medicine.Int.J. MediPharm Res. 2022,8(1),pp 1-21.
Abstract
The last decade has witnessed a paradigm shift in genetic medicine, transitioning the field of gene editing and gene therapy from a conceptual framework to a robust clinical reality. Innovations spanning
editing precision (e.g., base and prime editing), enhanced delivery mechanisms (e.g., next-generation AAV vectors and lipid nanoparticles), and expansion of therapeutic targets have accelerated this progress. This review critically examines the technological advancements underpinning this transition and highlights their transformative clinical applications across inherited genetic disorders, oncology, and infectious and immune diseases. Specifically, we discuss the evolution of CRISPR-Cas systems and their competitors, the optimization of in vivo and ex vivo delivery platforms, and the emerging success of treatments like CAR T-cell therapy and liver editing. While the potential of gene therapy as a pillar of precision medicine is undeniable, its equitable and safe deployment hinges on continued technological refinement, robust clinical trial design, and the parallel development of comprehensive ethical and regulatory frameworks to manage accessibility and long-term safety.
Keywords
Gene editing; CRISPR-Cas systems; base editing; prime editing; CAR-T therapy; gene delivery; offtarget effects; epigenetic editing; Wiskott-Aldrich syndrome; sickle cell disease; rare genetic disorders; immune therapy; precision medicine; clinical translation