Advances in Gene Therapy Research: From Discovery to Drug Candidates

Dear Colleagues,

Gene therapy translation from discovery to clinical testing has unique complexities and challenges. The progress in this field has been marked by a significant increase in the number of approved gene therapies in the last decade. The first Crispr-based gene therapy for sickle cell disease has been approved last year, marking a milestone for the field. It is commonly acknowledged that for many researchers and sponsors, gene therapy development must address many intricacies, including the selection of vectors and genes of interest, the creation of disease animal models that best mimic a genetic disease, the manufacturing of the gene therapies at the quality and scale required for clinical testing, and understanding an array of regulatory requirements.

With a focus on understanding the landscape and changing scenarios of translational research in gene therapy, this Special Issue of IJMS will cover developments on Lentiviral Vectors (CAR T cells, TCR cells, NK cells, CD34 cells, and others), Adeno-Associated Virus (AAV) vectors, and gene-editing tools (ZFN, Crispr, ASOs, and prime editors), from discovery through IND-enabling studies (CMC, efficacy, and toxicology studies) to drug candidate testing. Finally, for a broad view of the current gene therapy landscape, this Special Issue will include articles on different facets of gene therapy translation.

This Special Issue is supervised by Dr. Elizabeth A. Ottinger and Dr. Rodica Stan, assisted by our Guest Editor’s assistant editor Dr. Venkata Mangalampalli (National Institutes of Health).

Dr. Elizabeth Ottinger
Dr. Rodica Stan
Guest Editors

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• gene therapy

• gene editing
• viral vector
• preclinical development
• translational research