Results 31 to 40 of about 1,339,121 (337)

CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

Molecular Therapy: Methods & Clinical Development, 2021
Genome editing is potentially a curative technique available to all individuals with β-hemoglobinopathies, including sickle cell disease (SCD). Fetal hemoglobin (HbF) inhibits sickle hemoglobin (HbS) polymerization, and it is well described that ...
Selami Demirci, Alexis Leonard, Khaled Essawi, J. Tisdale   +3 more
semanticscholar   +1 more source

Candidate sequence variants and fetal hemoglobin in children with sickle cell disease treated with hydroxyurea. [PDF]

PLoS ONE, 2013
Fetal hemoglobin level is a heritable complex trait that strongly correlates swith the clinical severity of sickle cell disease. Only few genetic loci have been identified as robustly associated with fetal hemoglobin in patients with sickle cell disease,
Nancy S Green, Katherine L Ender, Farzana Pashankar, Catherine Driscoll, Patricia J Giardina, Craig A Mullen, Lorraine N Clark, Deepa Manwani, Jennifer Crotty, Sergey Kisselev, Kathleen A Neville, Carolyn Hoppe, Sandra Barral   +12 more
doaj   +1 more source

Gene Expression Analysis Of The Brazilian Type Of Hereditary Persistence Of Fetal Hemoglobin: Identification Of Genes That Could Be Related To γ-globin Activation

, 2015
Increased γ-globin production and consequent fetal hemoglobin (Hb F, α2γ2) formation is an important modulator of the clinical and hematological features of hemolytic anemias, such as sickle cell disease and β-thalassemia (β-thal).
Roversi F.M., Da Cunha A.F., De Albuquerque D.M., MacHado-Neto J.A., Da Costa F.F., Brugnerotto A.F., Lanaro C., Olalla Saad S.T., Carazzolle M.F.   +8 more
core   +3 more sources

Perspective: A novel prognostic for sickle cell disease

Saudi Journal of Medicine and Medical Sciences, 2018
Sickle hemoglobin (α2βS2) polymerization drives disease pathophysiology in sickle cell anemia. Fetal hemoglobin (α2γ2) restricts disease severity by inhibiting the polymerization of sickle hemoglobin in a concentration-dependent manner. Clinical decision-
Brian M Mozeleski, Abdullah Al-Rubaish, Amein Al-Ali, Jose Romero   +3 more
doaj   +1 more source

Fetomaternal hemorrhage - case report

Journal of Education, Health and Sport, 2022
Maternal fetal leakage (FMH) is a well-known cause of fetal anemia. We define it as the transition of the fetal erythrocytes into the maternal circulation. Its severity is determined by measuring the level of fetal hemoglobin in the mother's blood using
Jakub Gruszka, Anna Zwierzyńska, Magdalena Dolecka-Ślusarczyk, Olga Adamczyk-Gruszka   +3 more
doaj   +1 more source

Fetal Hemoglobin in Sickle Cell Anemia.

Blood, 2020
Fetal hemoglobin (HbF) can blunt the pathophysiology, temper the clinical course and offer prospects for curative therapy of sickle cell disease. This review focuses on: 1.
M. Steinberg
semanticscholar   +1 more source

Genetic association of fetal-hemoglobin levels in individuals with sickle cell disease in Tanzania maps to conserved regulatory elements within the MYB core enhancer. [PDF]

, 2015
BACKGROUND: Common genetic variants residing near upstream regulatory elements for MYB, the gene encoding transcription factor cMYB, promote the persistence of fetal hemoglobin (HbF) into adulthood. While they have no consequences in healthy individuals,
Soka, Deogratius, Harvest Mariki, Menzel, Stephan, Jeffrey C Barrett, Mariki, Harvest, Helen Rooks, Bruno Mmbando, Menzel, Stephan; id_orcid, Mmbando, Bruno, Tarjinder Singh, Stephan Menzel, Barrett, Jeffrey C., Sharon E Cox, Singh, Tarjinder, Mtatiro, Siana N, Barrett, Jeffrey C, Thein, Swee Lay, Deogratius Soka, Swee Lay Thein, Julie Makani, Mtatiro, Siana N., Siana N Mtatiro, Cox, Sharon E, Rooks, Helen, Mgaya, Josephine, Makani, Julie, Josephine Mgaya, Cox, Sharon E.   +27 more
core   +2 more sources

Effect of hydroxyurea on G gamma chain fetal hemoglobin synthesis by sickle-cell disease patients

Brazilian Journal of Medical and Biological Research, 2003
Hydroxyurea is used for sickle-cell disease patients in order to increase fetal hemoglobin synthesis and consequently decrease the severity of pain episodes.
S.M. Teixeira, L.C. Cortellazzi, H.Z.W. Grotto   +2 more
doaj   +1 more source

Induction of fetal hemoglobin: Lentiviral shRNA knockdown of HBS1L in β0-thalassemia/HbE erythroid cells.

PLoS ONE, 2023
Imbalanced globin chain output contributes to thalassemia pathophysiology. Hence, induction of fetal hemoglobin in β-thalassemia and other β-hemoglobinopathies are of continuing interest for therapeutic approaches.
Sukanya Chumchuen, Orapan Sripichai, Natee Jearawiriyapaisarn, Suthat Fucharoen, Chayanon Peerapittayamongkol   +4 more
doaj   +1 more source

The LSD1 inhibitor RN-1 recapitulates the fetal pattern of hemoglobin synthesis in baboons (P. anubis)

Haematologica, 2016
Increased fetal hemoglobin levels lessen the severity of symptoms and increase the lifespan of patients with sickle cell disease. Hydroxyurea, the only drug currently approved for the treatment of sickle cell disease, is not effective in a large ...
Angela Rivers, Kestis Vaitkus, Vinzon Ibanez, Maria Armila Ruiz, Ramasamy Jagadeeswaran, Yogen Saunthararajah, Shuaiying Cui, James D. Engel, Joseph DeSimone, Donald Lavelle   +9 more
doaj   +1 more source