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| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287278 | PMC |
| http://dx.doi.org/10.1073/pnas.2412536121 | DOI Listing |
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Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Lancet
December 2025
Biomedical Innovation Unit, Center for Research on Energy, Environment and Technology (CIEMAT), Madrid, Spain; Biomedical Network Research Center for Rare Diseases (CIBERER), Madrid, Spain; Sanitary Research Institute Fundación Jiménez Díaz (U.A.M), Madrid, Spain. Electronic address:
Background: Allogeneic haematopoietic stem-cell transplantation is the standard treatment for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation-associated complications such as an increased incidence of subsequent cancer are frequent. The aim of this study was to evaluate the safety and efficacy of the infusion of autologous gene-corrected haematopoietic stem cells as an alternative therapy for these patients.
Methods: This was an open-label, investigator-initiated phase 1/2 clinical trial (FANCOLEN-1) and long-term follow-up trial (up to 7 years post-treatment) in Spain.
Correction: Cost-efectiveness of robotic vs laparoscopic distal pancreatectomy. Results from the national prospective trial ROBOCOSTES.
Surg Endosc
November 2024
Hepato Pancreato Biliary Unit, Hospital del Mar, Pompeu Fabra University, Barcelona, Spain.
Genetic homogeneity and weak signatures of local adaptation in the marine mussel Mytilus chilensis.
Sci Rep
September 2024
Departamento de Biología Marina, Facultad de Ciencias del Mar, Universidad Católica del Norte, Coquimbo, Chile.
Article Synopsis
- Researchers studied the genetic structure and local adaptation of the marine mussel Mytilus chilensis in northern Patagonia, analyzing 5963 SNPs from 125 individuals over two years.
- They found high genetic diversity and low differentiation among populations, indicating that neutral processes, rather than strong local adaptation, shape the species' genetic makeup.
- The study also revealed low probabilities of accurately assigning individuals to their population of origin, suggesting that high dispersal and translocation in aquaculture hinder the development of distinct local genetic adaptations.
Consumers often inadvertently misperceive the health hazards associated with over-the-counter pesticides under the current textual labeling policy, potentially leading to improper use. We conducted an incentivized framed field experiment with eye tracking to evaluate the effectiveness of the current pesticide labels that convey risk using signal words (Caution, Warning, Danger) compared to two visually focused label alternatives: traffic light colors and skull intensity symbols. A total of 166 participants were randomly assigned to one of three label formats and asked to rank toxicity levels and make purchasing decisions within multiple price lists.
View Article and Find Full Text PDFNocturnal Glucose Profile According to Timing of Dinner Rapid Insulin and Basal and Rapid Insulin Type: An Connected Insulin Cap-Based Real-World Study.
Biomedicines
July 2024
Endocrinology and Nutrition Service, Hospital de la Santa Creu i Sant Pau, Institut de Recerca, 08041 Barcelona, Spain.
Background: A study to assess the glucose levels of people with type 1 diabetes (T1D) overnight, based on the insulin type and timing.
Methods: A real-world, retrospective study of T1D, using multiple daily insulin injections. Continuous glucose monitoring and insulin injection data were collected for ten hours after dinner using the connected cap.
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