Results 181 to 190 of about 1,398,221 (253)

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

open access: yesBritish Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi   +3 more
wiley   +1 more source

Iron, Tuberculosis, and Particle-Related Exposures Including Smoking. [PDF]

open access: yesPathogens
Ghio AJ   +4 more
europepmc   +1 more source

Identification of predictive factors for reversal of cerebral vasculopathy in an original longitudinal cohort study in newborns with sickle cell anaemia

open access: yesBritish Journal of Haematology, EarlyView.
Summary Cerebral macrovasculopathy (CV) is a major complication in children with sickle cell anaemia (SCA) and usually requires a long‐term transfusion programme (TP) to prevent stroke. This study aimed to identify factors predicting reversal of CV on TP in a single‐centre newborn cohort. Among 375 patients, 50 presented CV and received TP.
Julie Sommet   +16 more
wiley   +1 more source

A Narrative Review of Zinc Deficiency-Associated Anemia. [PDF]

open access: yesHematol Rep
Basmajian K   +13 more
europepmc   +1 more source

Functional assessment of inherited myeloid neoplasm‐associated SAMD9L germline variants via Monoallelic CRISPR modelling

open access: yesBritish Journal of Haematology, EarlyView.
Summary While the majority of myeloid neoplasms are sporadic, the increasing application of germline genetic testing has led the World Health Organization to designate ‘Myeloid malignancies with germline predisposition’ as a distinct clinical entity, carrying major implications for clinical care and research.
Elena Vuelta   +9 more
wiley   +1 more source

Novel approaches for drug development against chronic primary pain: A systematic review

open access: yesBritish Journal of Pharmacology, EarlyView.
Abstract Chronic primary pain (CPP) persisting for more than 3 months, associated with significant emotional distress without any known underlying cause, is an unmet medical need. Traditional or adjuvant analgesics do not provide satisfactory pain relief for a great proportion of these patients.
Valéria Tékus   +5 more
wiley   +1 more source

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