Aylin C Bonifacino
Overview
Explore the profile of Aylin C Bonifacino including associated specialties, affiliations and a list of published articles.
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27
Citations
580
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Recent Articles
1.
Demirci S, Zeng J, Palchaudhuri R, Wu C, Abraham D, Hayal T, et al.
Cell Stem Cell
. 2024 Dec;
32(2):209-226.e8.
PMID: 39642886
Editing the +58 region of the BCL11A erythroid enhancer has shown promise in treating β-globin disorders. To address variations in fetal hemoglobin (HbF) response, we investigated editing both +58 and ...
2.
Wang L, Suryawanshi G, Kim S, Guan X, Bonifacino A, Metzger M, et al.
Heliyon
. 2023 Oct;
9(9):e19435.
PMID: 37810095
Selective T-cell depletion prior to cell or organ transplantation is considered a preconditioning regimen to induce tolerance and immunosuppression. An immunotoxin consisting of a recombinant anti-CD3 antibody conjugated with diphtheria...
3.
Essawi K, Hakami W, Khan M, Martin R, Zeng J, Chu R, et al.
Mol Ther Methods Clin Dev
. 2023 Jun;
29:483-493.
PMID: 37273902
CRISPR-Cas9-based therapeutic genome editing approaches hold promise to cure a variety of human diseases. Recent findings demonstrate pre-existing immunity for the commonly used Cas orthologs from (SpCas9) and (SaCas9) in...
4.
Peslak S, Demirci S, Chandra V, Ryu B, Bhardwaj S, Jiang J, et al.
Mol Ther Nucleic Acids
. 2023 Feb;
31:452-465.
PMID: 36852088
Transcriptional enhancers can be in physical proximity of their target genes via chromatin looping. The enhancer at the β-globin locus (locus control region [LCR]) contacts the fetal-type () and adult-type...
5.
Uchida N, Li L, Nassehi T, Drysdale C, Yapundich M, Gamer J, et al.
Cell Rep Med
. 2021 May;
2(4):100247.
PMID: 33948577
Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells...
6.
Uchida N, Ferrara F, Drysdale C, Yapundich M, Gamer J, Nassehi T, et al.
Sci Transl Med
. 2021 Apr;
13(591).
PMID: 33910976
Hematopoietic stem cell gene therapy for hemoglobin disorders, including sickle cell disease, requires high-efficiency lentiviral gene transfer and robust therapeutic globin expression in erythroid cells. Erythropoietin is a key cytokine...
7.
Stringaris K, Hoyt R, Davidson-Moncada J, Pantin J, Tisdale J, Uchida N, et al.
Blood Adv
. 2020 Dec;
4(24):6148-6156.
PMID: 33351110
Intrabone (IB) injection of umbilical cord blood has been proposed as a potential mechanism to improve transplant engraftment and prevent graft failure. However, conventional IB techniques produce low retention of...
8.
de Ravin S, Brault J, Meis R, Li L, Theobald N, Bonifacino A, et al.
Blood Adv
. 2020 Dec;
4(23):5976-5987.
PMID: 33284949
Granulocytes from patients with chronic granulomatous disease (CGD) have dysfunctional phagocyte reduced nicotinamide adenine dinucleotide phosphate (NADPH) oxidase that fails to generate sufficient antimicrobial reactive oxidative species. CGD patients with...
9.
Demirci S, Zeng J, Wu Y, Uchida N, Shen A, Pellin D, et al.
J Clin Invest
. 2020 Sep;
130(12):6677-6687.
PMID: 32897878
Gene editing of the erythroid-specific BCL11A enhancer in hematopoietic stem and progenitor cells (HSPCs) from patients with sickle cell disease (SCD) induces fetal hemoglobin (HbF) without detectable toxicity, as assessed...
10.
Uchida N, Hsieh M, Raines L, Haro-Mora J, Demirci S, Bonifacino A, et al.
Nat Commun
. 2019 Oct;
10(1):4479.
PMID: 31578323
Hematopoietic stem cell (HSC) gene therapy is being evaluated for hemoglobin disorders including sickle cell disease (SCD). Therapeutic globin vectors have demanding requirements including high-efficiency transduction at the HSC level...