Over the past years, many significant advances have been made in the field of gene therapy and shown promising results in clinical trials conducted. Gene therapy aims at modifying or replacing a defective, inefficient, or nonfunctional gene with a healthy, functional gene by administration of genome material into the cell to cure genetic diseases. Various methods have been devised to do this by using several viral and non-viral vectors which are either administered by in vivo or ex vivo technique. Viral vectors are best suitable for this therapy due to their potential to invade cells and deliver their genetic material whereas non-viral vectors are less efficient than viral vectors but possess some advantages such as less immunogenic response and large gene carrying capacity. Recent advances in biotechnology such as CRISPR-Cas9 mediated genome engineering and Cancer treatment with Chimeric antigen receptor (CAR) T-cell therapy are addressed in this review. This review article also delves into some recent research studies, gene therapy trials, and its applications, laying out future hopes for gene therapy in the treatment of various diseases namely haemophilia, Muscular dystrophy, SCID, Sickle cell disease, Familial Hypercholesterolemia, Cystic Fibrosis. Additionally, it also includes various nanoformulations and clinical trial data related to gene therapy.

Mokshit Bhagat

Raj Kamal

Jyoti Sharma

Kirandeep Kaur

Amit Sharma

Gurjeet Singh Thakur

Rohit Bhatia

Ankit Awasthi

AAPS PharmSciTech

AAPS PHARMSCITECH 是一份经过同行评审的在线期刊，致力于为那些对药物剂型和递送系统的研究、开发和评估感兴趣的药物科学家和工程师提供服务，包括来自生物技术的药物和与商业化有关的制造科学这种剂型。由于其电子性质，AAPS PHARMSCITECH 渴望利用不断发展的电子技术来实现更快、更多样化的信息传递机制。欢迎提交不请自来的专家评论和研究文章。

AAPS PharmSciTech is a peer-reviewed online journal dedicated to serving pharmaceutical scientists and engineers interested in the research, development, and evaluation of drug formulations and delivery systems, including those related to biotechnology and manufacturing sciences associated with commercialization. Due to its electronic nature, AAPS PharmSciTech aims to leverage evolving electronic technologies to facilitate faster and more diverse information dissemination. We welcome unsolicited expert reviews and research articles.

AAPS PharmSciTech 是一本经过同行评审的在线期刊，专注于药物剂型和递送系统的研究、开发与评估，服务于药物科学家和工程师，尤其是涉及生物技术药物和商业化制造科学的领域。由于其电子出版的特性，AAPS PharmSciTech 希望利用不断发展的电子技术，实现更快速和多样化的信息传播。我们欢迎专家提交评论和研究文章。

Category	Quartile
医学	4
医学, 药学	4

Gene Therapy: Towards a New Era of Medicine

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Journal Info

Category	Quartile
PHARMACOLOGY & PHARMACY	2