Results 111 to 120 of about 70,815 (255)
Gene Editing for Haemophilia—The Next Frontier
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti +3 more
wiley +1 more source
Seventh Åland Island Meeting on von Willebrand Disease
ABSTRACT Introduction The seventh Åland Island Meeting on von Willebrand Disease (VWD) was held on the Åland archipelago in Finland, from 26 to 28 September 2024. Aim The meeting brought together experts in the field of VWD from around the world to share the latest advances and knowledge in VWD.
Riitta Lassila +17 more
wiley +1 more source
Frailty phenotype in adults with sickle cell disease. [PDF]
Oyedeji CI +15 more
europepmc +1 more source
ABSTRACT Introduction Patients with inherited bleeding and haemoglobin disorders face barriers to accessing timely dental care, increasing the risk of untreated oral disease and complications related to invasive procedures. Aim To evaluate the agreement between smartphone‐based asynchronous teledentistry and face‐to‐face examination for oral conditions,
Victor Cordeiro da Silva +7 more
wiley +1 more source
Effectiveness of health care transition interventions for sickle cell disease. [PDF]
Azul M +14 more
europepmc +1 more source
ABSTRACT Aim Hypoxic preconditioning of cells holds promise for regenerative therapies, yet identifying effective and safe methods for clinical application remains challenging. We aimed to determine optimal hypoxia‐mimetic small molecules (SMs) that stabilize hypoxia‐inducible factor‐1α (HIF‐1α) and their dosages for hypoxic preconditioning in stem ...
Hong Wang +9 more
wiley +1 more source
Should Sickle Cell Disease Be Considered a Cancer Predisposition Syndrome? [PDF]
Casadessus E, Pastore Y, Pincez T.
europepmc +1 more source
Determinant Factors of Self‐Care Deficit in People With Chronic Wounds: A Scoping Review
ABSTRACT Aims To identify and synthesise the scientific evidence on determinants of self‐care deficits in people with chronic wounds. Design Scoping review based on the Joanna Briggs Institute guidelines. Methods This scoping review was conducted by a review team responsible for study screening, data extraction and synthesis.
Janislei Soares Dantas +4 more
wiley +1 more source
Transformative therapies for sickle cell disease. [PDF]
Jamani K +3 more
europepmc +1 more source

