End points for sickle cell disease clinical trials: Renal and cardiopulmonary, cure, and low-resource settings.
Farrell, AT; Panepinto, J; Desai, AA; Kassim, AA; Lebensburger, J; Walters, MC; Bauer, DE; Blaylark, RM; DiMichele, DM; Gladwin, MT; et al.
Blood Advances.
2019;
3:4002-4020.
CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sites.
Jayavaradhan, R; Pillis, DM; Goodman, M; Zhang, F; Zhang, Y; Andreassen, PR; Malik, P.
Nature Communications.
2019;
10.
Role of the coagulation system in the pathogenesis of sickle cell disease.
Nasimuzzaman, M; Malik, P.
Blood Advances.
2019;
3:3170-3180.
Robust clinical and laboratory response to hydroxyurea using pharmacokinetically guided dosing for young children with sickle cell anemia.
McGann, PT; Niss, O; Dong, M; Marahatta, A; Howard, TA; Mizuno, T; Lane, A; Kalfa, TA; Malik, P; Quinn, CT; et al.
American Journal of Hematology.
2019;
94:871-879.
Abnormal submaximal cardiopulmonary exercise parameters predict impaired peak exercise performance in sickle cell anemia patients.
Powell, AW; Alsaied, T; Niss, O; Fleck, RJ; Malik, P; Quinn, CT; Mays, WA; Taylor, MD; Chin, C.
Pediatric Blood and Cancer.
2019;
66.
Elimination of the fibrinogen integrin aMb2-binding motif improves renal pathology in mice with sickle cell anemia.
Nasimuzzaman, M; Arumugam, PI; Mullins, ES; James, JM; VandenHeuvel, K; Narciso, MG; Shaw, MA; McGraw, S; Aronow, BJ; Malik, P.
Blood Advances.
2019;
3:1519-1532.
Therapeutic strategies for sickle cell disease: towards a multi-agent approach.
Telen, MJ; Malik, P; Vercellotti, GM.
Nature Reviews Drug Discovery.
2019;
18:139-158.
A Versatile Tool for the Quantification of CRISPR/Cas9-Induced Genome Editing Events in Human Hematopoietic Cell Lines and Hematopoietic Stem/Progenitor Cells.
Jayavaradhan, R; Pillis, DM; Malik, P.
Journal of Molecular Biology.
2019;
431:102-110.
Diastolic dysfunction is associated with exercise impairment in patients with sickle cell anemia.
Alsaied, T; Niss, O; Powell, AW; Fleck, RJ; Cnota, JF; Chin, C; Malik, P; Quinn, CT; Taylor, MD.
Pediatric Blood and Cancer.
2018;
65:e27113-e27113.
Angiotensin receptor signaling in sickle cell anemia has a reno-protective effect on urine concentrating ability but results in sickle glomerulopathy.
Roy, S; Rai, P; Mpollo, ME M; Chang, K; Rizvi, T; Shanmukhappa, SK; VandenHeuvel, K; Aronow, B; Inagami, T; Cancelas, JA; et al.
American Journal of Hematology.
2018;
93:E177-E181.