Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of LAD-1.
Smith, RH; Bloomer, H; Fink, D; Keyvanfar, K; Nasimuzzaman, M; Sancheznieto, F; Dutta, R; Guenther Bui, K; Alvarado, LJ; Bauer, TR; et al.
Human Gene Therapy.
2022.
NRASQ61R mutation in human endothelial cells causes vascular malformations.
Boscolo, E; Pastura, P; Schrenk, S; Goines, J; Kang, R; Pillis, D; Malik, P; Le Cras, TD.
Angiogenesis.
2022;
25:331-342.
Differential CXCR4 expression on hematopoietic progenitor cells versus stem cells directs homing and engraftment.
Felker, S; Shrestha, A; Bailey, J; Pillis, DM; Siniard, D; Malik, P.
JCI insight.
2022;
7.
Successful use of veno-venous extracorporeal membrane oxygenation for acute chest syndrome in a child with sickle cell disease and SARS-CoV-2.
Koh, W; Malik, P; Whitehead, J; Morales, DL S; Hayes, D.
Pediatric Pulmonology.
2022;
57:1096-1099.
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI).
Adair, JE; Androski, L; Bayigga, L; Bazira, D; Brandon, E; Dee, L; Deeks, S; Draz, M; Dubé, K; Dybul, M; et al.
Gene Therapy.
2021.
Rapid and automated quantitation of dense red blood cells: A robust biomarker of hydroxyurea treatment response.
Sadaf, A; Quinn, CT; Korpik, JB; Pfeiffer, A; Reynaud, M; Niss, O; Malik, P; Ware, RE; Kalfa, TA; McGann, PT.
Blood Cells, Molecules, and Diseases.
2021;
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Early initiation of hydroxyurea (hydroxycarbamide) using individualised, pharmacokinetics-guided dosing can produce sustained and nearly pancellular expression of fetal haemoglobin in children with sickle cell anaemia.
Quinn, CT; Niss, O; Dong, M; Pfeiffer, A; Korpik, J; Reynaud, M; Bonar, H; Kalfa, TA; Smart, LR; Malik, P; et al.
British Journal of Haematology.
2021;
194:617-625.
Cardiac pathophysiology in sickle cell disease.
Gbotosho, OT; Taylor, M; Malik, P.
Journal of Thrombosis and Thrombolysis.
2021;
52:248-259.
Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience.
Karkoska, K; Todd, K; Niss, O; Clapp, K; Fenchel, L; Kalfa, TA; Malik, P; Quinn, CT; Ware, RE; McGann, PT.
Pediatric Blood and Cancer.
2021;
68.
FT-4202, an oral PKR activator, has potent antisickling effects and improves RBC survival and Hb levels in SCA mice.
Shrestha, A; Chi, M; Wagner, K; Malik, A; Korpik, J; Drake, A; Fulzele, K; Guichard, S; Malik, P.
Blood Advances.
2021;
5:2385-2390.