Punam Malik, MD

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 (Basingstoke). 2023; 30:216-221.

Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. McDonald, CL; Qasba, P; Anderson, DG; Bao, G; Colvin, RA; Kohn, DB; Malik, P; Mitchell, MJ; Pu, WT; Rawlings, DJ; et al. Human Gene Therapy. 2023; 34:83-89.

Preclinical Evaluation of Foamy Virus Vector-Mediated Gene Addition in Human Hematopoietic Stem/Progenitor Cells for Correction of Leukocyte Adhesion Deficiency Type 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; 33:1293-1304.

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:e151847.

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.

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; 90:102576.

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:e29008.