Results 191 to 200 of about 136,712 (243)

Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders. [PDF]

J Blood Med
Winkler R, Herath I, Kaczmarek R, Xiao W, Herzog RW, Severeid PE.   +5 more
europepmc   +1 more source

LDB1 represses fetal hemoglobin expression by enhancing BCL11A transcription. [PDF]

Redox Biol
Park SW, Choi CY, Park IB, Kang SJ, Lee HJ, Kim JI, Bae J, Geum D, Cheon YP, Chun T.   +9 more
europepmc   +1 more source

Transcriptomic data of piglet blood compartments with 3' mRNA sequencing. [PDF]

Data Brief
Payros G, Jason K, Gress L, Gusmini M, Liaubet L, Lippi Y.   +5 more
europepmc   +1 more source

Advances in Gene Therapy for Inherited Haemoglobinopathies. [PDF]

Hematol Rep
Gaspar AB, Gaspar HB.
europepmc   +1 more source

Single-cell transcriptomics reveals heterocellular γ-globin gene expression in Aγδβ-thalassemia. [PDF]

Blood Adv
Doerfler PA, Li Y, Yao Y, Feng R, Mayuranathan T, Zhang J, Kooienga E, Cullins DL, Jesudas R, Williams DC, Hankins JS, Cheng Y, Wood DK, Weiss MJ.   +13 more
europepmc   +1 more source

Profiling structural variations of the α-globin gene cluster by the single molecule real-time sequencing: remarkable diversity of the spectrum with rare and novel variants identified in a large Chinese cohort. [PDF]

Hum Genet
Wei D, Cheng Z, Wei W, Gui C, Liu J, Chen H, Ma Y, Peng X, Yu D, Huang Y, Lai Y, Gui B.   +11 more
europepmc   +1 more source

Functional correction and genome integrity with duplex base editing of β-thalassemic hematopoietic stem cells. [PDF]

Genome Biol
Papaioannou NY, Patsali P, Klermund J, Papasavva PL, Andrieux G, Koniali L, Naiisseh B, Christou S, Sitarou M, Kleanthous M, Cathomen T, Lederer CW.   +11 more
europepmc   +1 more source

Orai1 acts as a novel Ca²⁺ signal switch, balancing erythropoiesis through KLF1 regulation. [PDF]

Exp Mol Med
Lee YY, Koh H, Kim J, Kim SU, Lee JH, Park CY.   +5 more
europepmc   +1 more source

Enhanced induction of fetal hemoglobin by the combination of decitabine with RN-1 in β-thalassemia/HbE erythroid progenitor cells. [PDF]

Mol Med
Nualkaew T, Pongpaksupasin P, Munkongdee T, Buasuwan N, Paiboonsukwong K, Sripichai O, Engel JD, Hongeng S, Fucharoen S, Jearawiriyapaisarn N.   +9 more
europepmc   +1 more source