Current State of Human Gene Therapy: Approved Products and Vectors

In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments...

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Bibliographic Details
Published in:Pharmaceuticals (Basel, Switzerland) Switzerland), 2023-10, Vol.16 (10), p.1416
Main Authors: Shchaslyvyi, Aladdin Y, Antonenko, Svitlana V, Tesliuk, Maksym G, Telegeev, Gennadiy D
Format: Article
Language:English
Subjects:
Atherosclerosis
Cancer therapies
Cystic fibrosis
Drugs
FDA approval
Forecasts and trends
Gene expression
Gene therapy
gene therapy drugs
Genetic testing
human gene therapy
human gene therapy products
Lymphoma
Melanoma
Methods
MicroRNAs
non-viral vectors
Pancreatic cancer
physical delivery methods
Proteins
Review
viral vectors
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Summary:In the realm of gene therapy, a pivotal moment arrived with Paul Berg’s groundbreaking identification of the first recombinant DNA in 1972. This achievement set the stage for future breakthroughs. Conditions once considered undefeatable, like melanoma, pancreatic cancer, and a host of other ailments, are now being addressed at their root cause—the genetic level. Presently, the gene therapy landscape stands adorned with 22 approved in vivo and ex vivo products, including IMLYGIC, LUXTURNA, Zolgensma, Spinraza, Patisiran, and many more. In this comprehensive exploration, we delve into a rich assortment of 16 drugs, from siRNA, miRNA, and CRISPR/Cas9 to DNA aptamers and TRAIL/APO2L, as well as 46 carriers, from AAV, AdV, LNPs, and exosomes to naked mRNA, sonoporation, and magnetofection. The article also discusses the advantages and disadvantages of each product and vector type, as well as the current challenges faced in the practical use of gene therapy and its future potential.
ISSN:1424-8247
1424-8247
DOI:10.3390/ph16101416