| Texto completo | |
| Autor(es): Mostrar menos - |
Ros, Felipe Augusto
;
Couto, Samuel Campanelli Freitas
;
Milhomens, Jonathan
;
Ovider, Ian
;
Maio, Karina Tozatto
;
Jennifer, Viviane
;
Ramos, Rodrigo Nalio
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Picanco-Castro, Virginia
;
Kashima, Simone
;
Calado, Rodrigo T.
;
Barros, Luciana Rodrigues Carvalho
;
Rocha, Vanderson
Número total de Autores: 12
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| Tipo de documento: | Artigo Científico |
| Fonte: | CYTOTHERAPY; v. 25, n. 12, p. 7-pg., 2023-11-17. |
| Resumo | |
Background aims: Amidst the success of cell therapy for the treatment of onco-hematological diseases, the first recently Food and Drug Administration-approved gene therapy product for patients with transfusion-dependent ,B-thalassemia (TDT) indicates the feasibility of gene therapy as curative for genetic hematologic disorders. This work analyzed the current-world scenario of clinical trials involving gene therapy for ,B-hemoglobinopathies.Methods: Eighteen trials for patients with sickle cell disease (SCD) and 24 for patients with TDT were analyzed.Results: Most are phase 1 and 2 trials, funded by the industry and are currently recruiting volunteers. Treatment strategies for both diseases are fetal hemoglobin induction (52.4%); addition of wild-type or therapeutic ,B-globin gene (38.1%) and correction of mutations (9,5%). Gene editing (52.4%) and gene addition (40.5%) are the two most used techniques. The United States and France are the countries with the greatest number of clinical trials centers for SCD, with 83.1% and 4.2%, respectively. The United States (41.1%), China (26%) and Italy (6.8%) lead TDT trials centers.Conclusions: Geographic trial concentration indicates the high costs of this technology, logistical issues and social challenges that need to be overcome for gene therapy to reach low-and middle-income countries where SCD and TDT are prevalent and where they most impact the patient's health.(c) 2023 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved. (AU) | |
| Processo FAPESP: | 13/08135-2 - CTC - Centro de Terapia Celular |
| Beneficiário: | Dimas Tadeu Covas |
| Modalidade de apoio: | Auxílio à Pesquisa - Centros de Pesquisa, Inovação e Difusão - CEPIDs |
| Processo FAPESP: | 20/07055-9 - Núcleo de Terapia Celular - NuTeC |
| Beneficiário: | Rodrigo do Tocantins Calado de Saloma Rodrigues |
| Modalidade de apoio: | Auxílio à Pesquisa - Núcleos de Pesquisa Orientada a Problemas em São Paulo |
| Processo FAPESP: | 22/07503-7 - Indução de hemoglobina fetal como estratégia de tratamento para anemia falciforme utilizando terapia gênica |
| Beneficiário: | Felipe Augusto Rós |
| Modalidade de apoio: | Bolsas no Brasil - Doutorado |