Advances in Gene Therapy: Novel Strategies for Targeted Gene Delivery and Therapeutic Applications
PRANAV K JADHAV
, SHIVRAJ P JADHAV , SEJAL R PAGAR , VAIBHAV B GUNJAL , KIRTI G SONARE
Gene, development, CRISPR-Cas9, barriers, cancer, DNA, RNA
Gene therapy has emerged as a promising approach to treat genetic disorders and diseases over the last 25 years. This review explores its evolution, aims, and scope. Gene therapy corrects genetic abnormalities by introducing functional genes, potentially offering long-lasting therapeutic effects. Recent technological advancements enable researchers to target challenging organs and genetic anomalies untouched by traditional treatments. It has shown remarkable progress in treating neurodegenerative disorders and is integral to therapeutic strategies for inherited and acquired human diseases, potentially revolutionizing medicine with personalized treatments. Future research goals include identifying suitable targets, precise delivery methods, immune response management, regulatory control, and minimizing side effects for enhanced efficacy and safety. The ongoing progress and potential of gene therapy offer hope for improved patient health and quality of life. In the context of cancer treatment, gene therapy holds the potential to deliver essential proteins and regulate gene expression, potentially replacing traditional treatments for long-term benefits. While monogenetic diseases have seen success, challenges persist for common conditions like cystic fibrosis and muscular dystrophy. Modified adenoviruses are explored as effective gene delivery vehicles to overcome these challenges. Principles of chemistry, manufacturing, and control (CMC), along with quality assurance, are crucial for gene therapy product safety and efficacy during clinical trials. Gene therapy, combined with technologies like CRISPR-Cas9, presents exciting opportunities for precision medicine, marking a milestone in personalized and targeted healthcare.
"Advances in Gene Therapy: Novel Strategies for Targeted Gene Delivery and Therapeutic Applications", IJSDR - International Journal of Scientific Development and Research (www.IJSDR.org), ISSN:2455-2631, Vol.8, Issue 10, page no.9 - 15, October-2023, Available :https://ijsdr.org/papers/IJSDR2310002.pdf
Volume 8
Issue 10,
October-2023
Pages : 9 - 15
Paper Reg. ID: IJSDR_208772
Published Paper Id: IJSDR2310002
Downloads: 000347245
Research Area: Pharmacy
Country: CHANDWAD, MAHARASTRA, INDIA
ISSN: 2455-2631 | IMPACT FACTOR: 9.15 Calculated By Google Scholar | ESTD YEAR: 2016
An International Scholarly Open Access Journal, Peer-Reviewed, Refereed Journal Impact Factor 9.15 Calculate by Google Scholar and Semantic Scholar | AI-Powered Research Tool, Multidisciplinary, Monthly, Multilanguage Journal Indexing in All Major Database & Metadata, Citation Generator
Publisher: IJSDR(IJ Publication) Janvi Wave
