Advancements in Gene Therapy for Human Disease: Trend of Current Clinical Trials

M Delshad, Z Davoodi-Moghaddam, M Khademi… - European Journal of …, 2024 - Elsevier
In an era of rapid scientific advancement, gene therapy has emerged as a groundbreaking
approach with the potential to revolutionize the treatment of a myriad of diseases and …

Gene therapy for deafness: we can do more

Y Chen, J Zhong, Y Shu - Nature Reviews Genetics, 2024 - nature.com
Challenges in the development of treatments for hereditary hearing loss include the
exploration of the underlying pathological mechanisms, the comprehensive evaluation of …

Affordable pricing of CRISPR treatments is a pressing ethical imperative

J Rueda, Í de Miguel Beriain, L Montoliu - The CRISPR journal, 2024 - liebertpub.com
Casgevy, the world's first approved CRISPR-based cell therapy, has been priced at $2.2
million per patient. Although this hefty price tag was widely anticipated, the extremely high …

In vivo engineering chimeric antigen receptor immune cells with emerging nanotechnologies

H Ren, Y **, H Huang, W Wu, X Dai, W Fang, J Qin… - Nano Today, 2024 - Elsevier
Adoptive cell therapy with chimeric antigen receptor (CAR) has revolutionized cancer
treatment in the past decade. Now several adoptive cell therapies are approved, and …

Clinical trials to gene therapy development and production in Brazil: a review

JAM Saute, V Picanço-Castro… - The Lancet Regional …, 2025 - thelancet.com
Challenges related to the implementation of gene therapy products are daunting for low-to
middle-income countries, such as Brazil, and include the creation of an appropriate …

Sickle cell disease in India: the journey and hope for the future

K Gupta, L Krishnamurti, D Jain - Hematology, 2024 - ashpublications.org
India, the most populous nation in the world, also has a high frequency of the sickle
hemoglobin (HbS) allele globally. The Arab Indian HbS haplotype in India is characterized …

Strengthening advanced therapy for sickle cell disease in Africa: experience from sickle cell disease centre in Dar es Salaam, Tanzania

D Bukini, A Rifai, C Kanza, F Luoga, D Maingu… - BMJ Global …, 2025 - gh.bmj.com
Despite progress in healthcare services for individuals living with sickle cell disease (SCD)
in Africa, substantial gaps remain in advanced treatments for SCD. To help address this …

[HTML][HTML] Efficient manufacturing of CAR-T cells from whole blood: a scalable approach to reduce costs and enhance accessibility in cancer therapy

R Traynor, I Vignola, S Sarkar, M Prochazkova, Y Cai… - Cytotherapy, 2024 - Elsevier
Background Chimeric antigen receptor T (CAR-T) cells have significantly advanced the
treatment of cancers such as leukemia and lymphoma. Traditionally, T cells are collected …

Affordable pricing of CRISPR treatments is a pressing ethical imperative

J Rueda Etxebarria, Í Miguel Beriain, L Montoliu - 2024 - digibug.ugr.es
Casgevy, the world's first approved CRISPR-based cell therapy, has been priced at $2.2
million per patient. Although this hefty price tag was widely anticipated, the extremely high …

[PDF][PDF] The future of precision medicine: Integrating genetic engineering into pharmacological research

MM Hossen - 2025 - genesispcl.com
The integration of genetic engineering into pharmacological research is transforming the
landscape of precision medicine, offering the potential to revolutionize healthcare. Precision …