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Mark C Walters

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Articles 84
Citations 2347
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Recent Articles
1.
Kassim A, Walters M, Eapen M, Smith M, Logan B, Solh M, et al.
NEJM Evid . 2025 Feb; 4(3):EVIDoa2400192. PMID: 39998298
Background: Related human leukocyte antigen (HLA)-haploidentical bone marrow transplantation (BMT) with posttransplant cyclophosphamide may be curative for sickle cell disease. However, graft failure, severe graft-versus-host disease (GVHD), infections, and mortality...
2.
Kwiatkowski J, Walters M, Hongeng S, Yannaki E, Kulozik A, Kunz J, et al.
Lancet . 2024 Nov; 404(10468):2175-2186. PMID: 39527960
Background: Transfusion-dependent β-thalassaemia (TDT) is a severe disease, resulting in lifelong blood transfusions, iron overload, and associated complications. Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells...
3.
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, et al.
Blood Adv . 2024 Oct; 9(5):955-965. PMID: 39471440
Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2 years after biologic assignment...
4.
Lessard S, Rimmele P, Ling H, Moran K, Vieira B, Lin Y, et al.
Sci Rep . 2024 Oct; 14(1):24298. PMID: 39414860
BIVV003 is a gene-edited autologous cell therapy in clinical development for the potential treatment of sickle cell disease (SCD). Hematopoietic stem cells (HSC) are genetically modified with mRNA encoding zinc...
5.
John T, Walters M, Rangarajan H, Rahim M, McKinney C, Bollard C, et al.
Blood Adv . 2024 Oct; 8(23):6055-6063. PMID: 39374573
The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1507 leadership and the data safety monitoring board (DSMB) established incremental entry criteria for children aged 5 to 14.99 years...
6.
Braniecki S, Vichinsky E, Walters M, Shenoy S, Shi Q, Moore T, et al.
Front Neurol . 2024 Jun; 15:1263373. PMID: 38841694
Background: Due to the risk of cerebral vascular injury, children and adolescents with high-risk sickle cell disease (SCD) experience neurocognitive decline over time. Haploidentical stem cell transplantation (HISCT) from human...
7.
Frangoul H, Locatelli F, Sharma A, Bhatia M, Mapara M, Molinari L, et al.
N Engl J Med . 2024 Apr; 390(18):1649-1662. PMID: 38661449
Background: Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of...
8.
Eapen M, Kou J, Andreansky M, Bhatia M, Brochstein J, Chaudhury S, et al.
Am J Hematol . 2024 Feb; 99(4):785-788. PMID: 38343182
No abstract available.
9.
Locatelli F, Corbacioglu S, Hobbs W, Frangoul H, Walters M
Am J Hematol . 2023 Nov; 99(3):430-438. PMID: 38010293
A growing number of gene therapy- and gene editing-based treatments for patients with sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the underlying cause of...
10.
Olson T, Walters M
Lancet Haematol . 2023 Oct; 10(10):e798-e800. PMID: 37793770
No abstract available.