Publications by authors named "P Telfer"

Article Synopsis
  • Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
  • A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
  • Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.
View Article and Find Full Text PDF
Article Synopsis
  • - The acute pain crisis (APC) is the most common complication of sickle cell disease (SCD) and often requires strong opioid treatment and supportive care in hospitals.
  • - Despite the existence of guidelines for managing APC, these are frequently not followed, leading to suboptimal care in emergency departments and acute medical wards.
  • - The review suggests potential improvements in patient care, such as personalized care plans, quicker opioid delivery methods, and dedicated specialist units, but acknowledges that inadequate evidence supporting these approaches hinders their adoption in healthcare systems.
View Article and Find Full Text PDF

Sub-Saharan Africa is under-represented in global biodiversity datasets, particularly regarding the impact of land use on species' population abundances. Drawing on recent advances in expert elicitation to ensure data consistency, 200 experts were convened using a modified-Delphi process to estimate 'intactness scores': the remaining proportion of an 'intact' reference population of a species group in a particular land use, on a scale from 0 (no remaining individuals) to 1 (same abundance as the reference) and, in rare cases, to 2 (populations that thrive in human-modified landscapes). The resulting bii4africa dataset contains intactness scores representing terrestrial vertebrates (tetrapods: ±5,400 amphibians, reptiles, birds, mammals) and vascular plants (±45,000 forbs, graminoids, trees, shrubs) in sub-Saharan Africa across the region's major land uses (urban, cropland, rangeland, plantation, protected, etc.

View Article and Find Full Text PDF

Gene modification of haematopoietic stem cells (HSCs) is a potentially curative approach to sickle cell disease (SCD) and offers hope for patients who are not eligible for allogeneic HSC transplantation. Current approaches require in vitro manipulation of healthy autologous HSC prior to their transplantation. However, the health and integrity of HSCs may be compromised by a variety of disease processes in SCD, and challenges have emerged in the clinical trials of gene therapy.

View Article and Find Full Text PDF