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IJSDR
INTERNATIONAL JOURNAL OF SCIENTIFIC DEVELOPMENT AND RESEARCH
International Peer Reviewed & Refereed Journals, Open Access Journal
ISSN Approved Journal No: 2455-2631 | Impact factor: 8.15 | ESTD Year: 2016
open access , Peer-reviewed, and Refereed Journals, Impact factor 8.15

Issue: May 2024

Volume 9 | Issue 5

Impact factor: 8.15

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Paper Title: Advances in Gene Therapy: Novel Strategies for Targeted Gene Delivery and Therapeutic Applications
Authors Name: PRANAV K JADHAV , SHIVRAJ P JADHAV , SEJAL R PAGAR , VAIBHAV B GUNJAL , KIRTI G SONARE
Unique Id: IJSDR2310002
Published In: Volume 8 Issue 10, October-2023
Abstract: 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.
Keywords: Gene, development, CRISPR-Cas9, barriers, cancer, DNA, RNA
Cite Article: "Advances in Gene Therapy: Novel Strategies for Targeted Gene Delivery and Therapeutic Applications", International Journal of Science & Engineering Development Research (www.ijsdr.org), ISSN:2455-2631, Vol.8, Issue 10, page no.9 - 15, October-2023, Available :http://www.ijsdr.org/papers/IJSDR2310002.pdf
Downloads: 000338720
Publication Details: Published Paper ID: IJSDR2310002
Registration ID:208772
Published In: Volume 8 Issue 10, October-2023
DOI (Digital Object Identifier):
Page No: 9 - 15
Publisher: IJSDR | www.ijsdr.org
ISSN Number: 2455-2631

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