Gene Therapy & Genome Editing (BIO308) | 40+ Verified Q&As on CRISPR, Viral Vectors, Recombinant DNA

This document contains over 40 expertly verified questions and answers for the course Gene Therapy & Genome Editing (BIO308), focusing on cutting-edge genetic engineering techniques, their mechanisms, and applications. The content explores core concepts such as recombinant DNA technology, restriction enzymes, gene cloning, plasmid vectors, and therapeutic gene delivery using viral systems. A significant portion of the document is dedicated to CRISPR-Cas systems, explaining gene disruption via NHEJ (non-homologous end joining), gene addition through HDR (homology-directed repair), and their implications for treating both recessive and dominant genetic diseases. The difference between somatic and germ-line gene editing is clearly outlined, including ethical considerations tied to embryo modification and future generational impact. Key practical applications such as ex vivo gene therapy, biopharming, and the molecular tools used to alter genomic sequences are presented in clear, test-focused language. The questions simulate exam formats and include concise, high-yield answers aligned with academic and clinical learning outcomes. This resource is ideal for: Undergraduate students in Genetics, Molecular Biology, or Biomedical Sciences Final-year biology or pre-med students preparing for term exams or standardized tests Graduate students and researchers reviewing core gene-editing principles Biotechnology and pharmaceutical trainees seeking applied knowledge in gene therapy development Whether used for revision or concept reinforcement, this document offers a concise yet thorough overview of the essential principles of modern genetic modification. Keywords: gene therapy, genome editing, CRISPR, HDR, NHEJ, somatic gene editing, germ-line editing, recombinant DNA, plasmid vector, restriction enzymes, viral vector, cloning, guide RNA, ligase, genetic engineering, ex vivo therapy, biopharming