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Transcriptional regulators of fetal hemoglobin [PDF]

Hematology, Transfusion and Cell Therapy
Sickle cell anemia is a hereditary disease caused by sickle-shaped red blood cells that can lead to vaso-occlusive crises. Treatment options are currently limited, highlighting the need to develop new clinical approaches.
Gabriela Pereira dos Santos, Larissa Teodoro Rabi, André Alves Bezerra, Marcelo Rodrigues da Cunha, Amilton Iatecola, Victor Augusto Ramos Fernandes   +5 more
doaj   +4 more sources

Potent and uniform fetal hemoglobin induction via base editing. [PDF]

Nat Genet, 2023
A comparison of fetal hemoglobin gene editing strategies using human sickle cell disease donor cells and in vivo transplantation finds that adenine base editing of the –175A>G site in the γ-globin gene promoters results in durable and potent expression ...
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS.   +26 more
europepmc   +2 more sources

Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin

eLife, 2022
Naturally occurring point mutations in the HBG promoter switch hemoglobin synthesis from defective adult beta-globin to fetal gamma-globin in sickle cell patients with hereditary persistence of fetal hemoglobin (HPFH) and ameliorate the clinical severity.
Nithin Sam Ravi, Beeke Wienert, Stacia K Wyman, Henry William Bell, Anila George, Gokulnath Mahalingam, Jonathan T Vu, Kirti Prasad, Bhanu Prasad Bandlamudi, Nivedhitha Devaraju, Vignesh Rajendiran, Nazar Syedbasha, Aswin Anand Pai, Yukio Nakamura, Ryo Kurita, Muthuraman Narayanasamy, Poonkuzhali Balasubramanian, Saravanabhavan Thangavel, Srujan Marepally, Shaji R Velayudhan, Alok Srivastava, Mark A DeWitt, Merlin Crossley, Jacob E Corn, Kumarasamypet M Mohankumar   +24 more
doaj   +2 more sources

A Cell-Permeant Nanobody-Based Degrader That Induces Fetal Hemoglobin. [PDF]

ACS Cent Sci, 2022
Proximity-based strategies to degrade proteins have enormous therapeutic potential in medicine, but the technologies are limited to proteins for which small molecule ligands exist.
Shen F, Zheng G, Setegne M, Tenglin K, Izadi M, Xie H, Zhai L, Orkin SH, Dassama LMK.   +8 more
europepmc   +2 more sources

Hereditary persistence of fetal hemoglobin

Asian Journal of Transfusion Science, 2020
Hereditary persistence of fetal hemoglobin (HPFH) is a benign condition in which significant fetal hemoglobin production continues well into adulthood, disregarding the normal shutoff point after which only adult-type hemoglobin should be produced.
Dharmesh Chandra Sharma, Sachin Singhal, Poonam Woike, Sunita Rai, Mahesh Yadav, Rajesh Gaur   +5 more
doaj   +4 more sources

Fetal hemoglobin induction during decitabine treatment of elderly patients with high-risk myelodysplastic syndrome or acute myeloid leukemia: a potential dynamic biomarker of outcome

Haematologica, 2019
Hematologic responses to hypomethylating agents are often delayed in patients with myelodysplastic syndrome or acute myeloid leukemia. Fetal hemoglobin is a potential novel bio-marker of response: recently, we demonstrated that a high fetal hemoglobin ...
Julia Stomper, Gabriele Ihorst, Stefan Suciu, Philipp N. Sander, Heiko Becker, Pierre W. Wijermans, Christoph Plass, Dieter Weichenhan, Emmanuel Bissé, Rainer Claus, Michael Lübbert   +10 more
doaj   +2 more sources

A cellular reporter system to evaluate endogenous fetal hemoglobin induction and screen for therapeutic compounds [PDF]

HemaSphere
Reactivation of fetal hemoglobin expression alleviates the symptoms associated with β‐globinopathies, severe hereditary diseases with significant global health implications due to their high morbidity and mortality rates.
Thijs C. J. Verheul, Nynke Gillemans, Kerstin Putzker, Rezin Majied, Tingyue Li, Memnia Vasiliou, Bert Eussen, Annelies deKlein, Wilfred F. J. vanIJcken, Emile van denAkker, Marieke vonLindern, Joe Lewis, Ulrike Uhrig, Yukio Nakamura, Thamar vanDijk, Sjaak Philipsen   +15 more
doaj   +2 more sources

Agentes indutores da síntese de hemoglobina fetal Fetal hemoglobin inducing factors [PDF]

Revista Brasileira de Hematologia e Hemoterapia, 2007
Anemia falciforme é uma doença heterogênea caracterizada por uma grande variabilidade clínica. Desde as primeiras observações desta doença, foi visto que aumento no nível de hemoglobina fetal (HbF) estava associado com manifestações clínicas mais brandas,
Maria Stella Figueiredo
doaj   +4 more sources

Oxidative stress in preeclampsia and the role of free fetal hemoglobin

Frontiers in Physiology, 2015
Preeclampsia is a leading cause of pregnancy complications and affects 3–7 % of pregnant women. This review summarizes the current knowledge of a new potential etiology of the disease, with a special focus on hemoglobin-induced oxidative stress ...
Stefan Rocco Hansson, Åsa eNääv, Lena eErlandsson   +2 more
doaj   +2 more sources

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

Nature Communications, 2022
Sickle cell disease and β-thalassemia affect the production of the adult β-hemoglobin chain. The clinical severity is lessened by mutations that cause fetal γ-globin expression in adult life (i.e., the hereditary persistence of fetal hemoglobin ...
P. Antoniou, G. Hardouin, P. Martinucci, G. Frati, T. Felix, A. Chalumeau, L. Fontana, J. Martin, C. Masson, Mégane Brusson, G. Maule, M. Rosello, C. Giovannangeli, V. Abramowski, J. de Villartay, J. Concordet, Filippo Del Bene, W. El Nemer, Mario Amendola, M. Cavazzana, A. Cereseto, Oriana Romano, A. Miccio   +22 more
semanticscholar   +1 more source