Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors.
By Vijay Kumar MalesuNov 12 2023Reviewed by Sophia Coveney In a recent review published in Gene Therapy, a group of authors explored the progress and persistent hurdles in gene therapy for inherited blood disorders, malignancies via chimeric antigen receptors -T cells, and varied diseases treated with in vivo adeno-associated virus vectors, addressing the transformative cures and the economic and manufacturing complexities involved.
Advances in tissue typing, conditioning regimens, and supportive care have improved the outcomes of HSCT over the decades. However, the procedure's success is limited by the availability of matched donors and potential immunological complications. Gene therapy has also advanced in treating hemoglobinopathies like β-thalassemia and sickle cell disease, with novel vectors and gene editing techniques mitigating disease severity and improving patient outcomes.
Redirecting T-cell specificity Related StoriesTCRs leverage the natural antigen specificity of T cells. Isolation and expansion of TILs, or transgenic introduction of TCRs into non-tumor-specific T cells, have augmented anti-tumor responses. Enhancing T-cell potency through genetic modification The therapeutic potential of CAR-T cells depends on precise targeting, coverage of tumor antigens, and robust expansion.
The AAV cargo configuration Gene therapy applications necessitate the replacement of AAV's native genome with a therapeutic expression cassette containing the gene of interest. This cassette, bound by two Inverted Terminal Repeats and inclusive of a promoter and poly A signal, allows for targeted gene expression by selecting tissue-specific promoters to mitigate undesired immune reactions.
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