Base editing of HBG1 and HBG2 promoters for sickle cell disease (Registro nro. 22925)
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| 000 -Cabecera | |
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| Campo de control interno | 03457nam a2200337 4500 |
| 001 - Número de control | |
| control field | ESSALUD |
| 005 - Fecha y hora de la última transacción | |
| Campo de control | 20260903142036.0 |
| 007 - Tipo material - Descripcion fisica - info general | |
| Tipo material - Descripcion fisica - info general | ta |
| 008 - Elementos de Longitud Fija - Información General | |
| Elementos de Longitud Fija - Información | t pe ||||| |||| 00| 0 spa d |
| 040 ## - Origen de la Catalogación | |
| Origen de la Catalogación | BMG |
| 041 ## - Idioma | |
| Idioma | eng |
| 100 ## - Autor | |
| Autor | Gupta, Ashish O. |
| Rol del Autor | Autor |
| 9 (RLIN) | 54539 |
| 245 ## - Titulo | |
| Titulo | Base editing of HBG1 and HBG2 promoters for sickle cell disease |
| 300 ## - Páginas | |
| Paginación | páginas: 1824-1835 |
| 520 ## - Resumen | |
| Resumen | Background: Sickle cell disease is characterized by chronic hemolytic anemia and recurrent severe vaso-occlusive crises. Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited to target the HBG1 and HBG2 promoters and inhibit BCL11A binding without altering BCL11A expression, yielding a switch in hemoglobin production from sickle hemoglobin (HbS) to antisickling fetal hemoglobin (HbF). Methods: In this phase 1–2 study, we enrolled patients 12 to 35 years of age with sickle cell disease who had had at least four severe vaso-occlusive crises in the 2 years before enrollment. After myeloablative conditioning with pharmacokinetically guided administration of busulfan, patients received a single infusion of risto-cel (at a dose of ≥3.0×106 viable CD34+ cells per kilogram of body weight). The primary efficacy end point was freedom from severe vaso-occlusive crises for 12 consecutive months, starting later than 60 days after the last red-cell transfusion. This interim analysis was unplanned; here, we describe safety, editing, engraftment, and hemoglobin production and the number of severe vaso-occlusive crises starting later than 60 days after the last red-cell transfusion. Results: A total of 31 patients received risto-cel and were followed for a mean of 6.6 months (range, 0.3 to 20.4). A median of one cycle (range, one to five) was required for stem-cell collection. Neutrophil engraftment occurred at a median of 17.5 days, and platelet engraftment at a median of 19 days. One patient died from idiopathic pneumonia syndrome. All 31 patients had at least one adverse event, 27 (87%) had an adverse event of grade 3 or higher, and 12 (39%) had a serious adverse event. At 6 months, the mean fraction of on-target edited alleles in peripheral blood was 67.4%, the mean HbF as a fraction of total hemoglobin was more than 60%, and the HbS as a fraction of total hemoglobin was less than 40% (among 13 patients); these levels were maintained throughout follow-up. No investigator-reported severe vaso-occlusive crises occurred later than 60 days after the last red-cell transfusion. Conclusions: Treatment with risto-cel was followed by rapid engraftment and durable expression of HbF and reduction in HbS. These data support further investigation of risto-cel to treat sickle cell disease. |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | MEDICINA DEL ADOLESCENTE |
| 9 (RLIN) | 5533 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | ANEMIA |
| 9 (RLIN) | 7417 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | ENFERMEDADES INFANTILES |
| 9 (RLIN) | 53890 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | GENÉTICA GENERAL |
| 9 (RLIN) | 54107 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | HEMATOLOGÍA |
| 9 (RLIN) | 7422 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | ONCOLOGÍA GENERAL |
| 9 (RLIN) | 54224 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | CÉLULAS MADRE |
| 9 (RLIN) | 42759 |
| 700 ## - Autor Personal | |
| Autor Personal | Sharma, Akshay |
| 9 (RLIN) | 54540 |
| 700 ## - Autor Personal | |
| Autor Personal | Frangoul, Haydar |
| 9 (RLIN) | 54541 |
| 700 ## - Autor Personal | |
| Autor Personal | Kanter, Julie |
| 9 (RLIN) | 54542 |
| 700 ## - Autor Personal | |
| Autor Personal | Mapara, Markus Y. |
| 9 (RLIN) | 54543 |
| 700 ## - Autor Personal | |
| Autor Personal | Dalal, Jignesh |
| 9 (RLIN) | 54544 |
| 700 ## - Autor Personal | |
| Autor Personal | Alavi, Asif |
| 9 (RLIN) | 54545 |
| 773 0# - Revista (Relacion con el numero) | |
| Host Biblionumber | 22717 |
| Host Itemnumber | 22676 |
| Ciudad, Editorial | Massachusetts NEJM Group |
| Codigo barras item/ejemplar | NEJM015 |
| Titulo de la Revista | The New England Journal of Medicine |
| Número de control de registro | ESSALUD |
| ISSN | 0028-4793 |
| 942 ## - Elementos de Koha | |
| Tipo de Documento | Artículos |
| Fecha procesamiento | 2026-08-31 |
| Catalogador | sqb |
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