From mutational signatures to practice: Artificial intelligence-guided repurposing for blast crisis chronic myeloid leukemia. [PDF]
Karmakar R +3 more
europepmc +1 more source
Long‐read sequencing‐based atlas of tissue‐specific expression of DNM1L transcript variants
Targeted long‐read sequencing resolves full‐length DNM1L (Drp1) isoforms and reveals conserved, tissue‐specific expression patterns across human and mouse tissues. Functional assays show that Drp1 isoforms differ in their ability to drive mitochondrial fission, independent of abundance, with specific exons modulating activity.
Feng Yan +19 more
wiley +1 more source
The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care. [PDF]
Tardif M, Saby M, Forté S, Pincez T.
europepmc +1 more source
From hepatic to hematopoietic: LRH‐1's expanding cellular repertoire to the immune system
The nuclear receptor LRH‐1 is a well‐characterized regulator of endodermal tissue. Yet, increasing evidence indicates that LRH‐1, although expressed at low levels, is also a critical regulator of the hematopoietic system. LRH‐1 regulates the immune system by contributing to immune cell‐specific functions.
Lukas Meisinger +3 more
wiley +1 more source
What is the best path towards allogeneic transplantation in MDS and AML? A survey among German-spreaking centers for allogeneic hematopoietic stem cell transplantation. [PDF]
Krause SW +19 more
europepmc +1 more source
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
Exploring gene editing as a potential therapeutic strategy for hemophilia. [PDF]
Kumarasamy N, Balakrishnan B.
europepmc +1 more source
ABSTRACT Introduction RUNX1 is a commonly mutated transcriptional regulator of hematopoiesis in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Mutated RUNX1 (mRUNX1) may associate with cross‐lineage immunophenotypic aberrancy, presenting potential complications for blast lineage assignment at diagnosis. Methods Clinical and laboratory
Yi Han Xia, Eric McGinnis
wiley +1 more source
Complement activation in kidney transplantation. [PDF]
Kanbay M +7 more
europepmc +1 more source

