Punam Malik , MD

Peer Reviewed Publications

van der Loo, J C M; Swaney, W P; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; Hacein-Bey-Abina, S; Nordling, D L; Cavazzana-Calvo, M; Thrasher, A J; Williams, D A; Reeves, L; Malik, P (2012. ) Critical variables affecting clinical-grade production of the self-inactivating gamma-retroviral vector for the treatment of X-linked severe combined immunodeficiency.Gene therapy, , 19 (8 ) ,872-6 More Information

van der Loo, J C M; Swaney, W P; Grassman, E; Terwilliger, A; Higashimoto, T; Schambach, A; Baum, C; Thrasher, A J; Williams, D A; Nordling, D L; Reeves, L; Malik, P (2012. ) Scale-up and manufacturing of clinical-grade self-inactivating ?-retroviral vectors by transient transfection.Gene therapy, , 19 (3 ) ,246-54 More Information

Sundaram, Nambirajan; Bennett, Michael; Wilhelm, Jamie; Kim, Mi-Ok; Atweh, George; Devarajan, Prasad; Malik, Punam (2011. ) Biomarkers for early detection of sickle nephropathy.American journal of hematology, , 86 (7 ) ,559-66 More Information

Perumbeti, Ajay; Malik, Punam (2010. ) Therapy for beta-globinopathies: a brief review and determinants for successful and safe correction.Annals of the New York Academy of Sciences, , 1202 ,36-44 More Information

Sundaram, Nambirajan; Tailor, Anitaben; Mendelsohn, Laurel; Wansapura, Janaka; Wang, Xunde; Higashimoto, Tomoyasu; Pauciulo, Michael W; Gottliebson, William; Kalra, Vijay K; Nichols, William C; Kato, Gregory J; Malik, Punam (2010. ) High levels of placenta growth factor in sickle cell disease promote pulmonary hypertension.Blood, , 116 (1 ) ,109-12 More Information

Perumbeti, Ajay; Malik, Punam (2010. ) Genetic correction of sickle cell anemia and beta-thalassemia: progress and new perspective.TheScientificWorldJournal, , 10 ,644-54 More Information

Arumugam, Paritha; Malik, Punam (2010. ) Genetic therapy for beta-thalassemia: from the bench to the bedside.Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program, , 2010 ,445-50 More Information

Wang, Daren; Zhang, Wei; Kalfa, Theodosia A; Grabowski, Gregory; Davies, Stella; Malik, Punam; Pan, Dao (2009. ) Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome.Proceedings of the National Academy of Sciences of the United States of America, , 106 (47 ) ,19958-63 More Information

Arumugam, Paritha I; Higashimoto, Tomoyasu; Urbinati, Fabrizia; Modlich, Ute; Nestheide, Shawna; Xia, Ping; Fox, Catherine; Corsinotti, Andrea; Baum, Christopher; Malik, Punam (2009. ) Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.Molecular therapy : the journal of the American Society of Gene Therapy, , 17 (11 ) ,1929-37 More Information

Arumugam, Paritha I; Urbinati, Fabrizia; Velu, Chinavenmeni S; Higashimoto, Tomoyasu; Grimes, H Leighton; Malik, Punam (2009. ) The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity.PloS one, , 4 (9 ) ,e6995 More Information

Urbinati, Fabrizia; Arumugam, Paritha; Higashimoto, Tomoyasu; Perumbeti, Anil; Mitts, Kyle; Xia, Ping; Malik, Punam (2009. ) Mechanism of reduction in titers from lentivirus vectors carrying large inserts in the 3'LTR.Molecular therapy : the journal of the American Society of Gene Therapy, , 17 (9 ) ,1527-36 More Information

Perumbeti, Ajay; Higashimoto, Tomoyasu; Urbinati, Fabrizia; Franco, Robert; Meiselman, Herbert J; Witte, David; Malik, Punam (2009. ) A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.Blood, , 114 (6 ) ,1174-85 More Information

Williams, Jon P; Wu, Jianqiang; Johansson, Gunnar; Rizvi, Tilat A; Miller, Shyra C; Geiger, Hartmut; Malik, Punam; Li, Wenling; Mukouyama, Yoh-suke; Cancelas, Jose A; Ratner, Nancy (2008. ) Nf1 mutation expands an EGFR-dependent peripheral nerve progenitor that confers neurofibroma tumorigenic potential.Cell stem cell, , 3 (6 ) ,658-69 More Information

Gonsalves, Caryn Suzanne; Li, Chen; Malik, Punam; Tahara, Stanley M; Kalra, Vijay K (2015. ) Peroxisome Proliferator alpha mediated transcription of miR-301a and miR-454 and their host gene SKA2 regulate Endothelin-1 and PAI-1 expression in sickle cell disease.Bioscience reports, , More Information

Arumugam, Paritha I; Mullins, Eric S; Shanmukhappa, Shiva Kumar; Monia, Brett P; Loberg, Anastacia; Shaw, Maureen A; Rizvi, Tilat; Wansapura, Janaka; Degen, Jay L; Malik, Punam (2015. ) Genetic diminution of circulating prothrombin ameliorates multiorgan pathologies in sickle cell disease mice.Blood, , 126 (15 ) ,1844-55 More Information

Gonsalves, Caryn S; Li, Chen; Mpollo, Marthe-Sandrine Eiymo Mwa; Pullarkat, Vinod; Malik, Punam; Tahara, Stanley M; Kalra, Vijay K (2015. ) Erythropoietin-mediated expression of placenta growth factor is regulated via activation of hypoxia-inducible factor-1? and post-transcriptionally by miR-214 in sickle cell disease.The Biochemical journal, , 468 (3 ) ,409-23 More Information

Carmo, Marlene; Risma, Kimberly A; Arumugam, Paritha; Tiwari, Swati; Hontz, Adrianne E; Montiel-Equihua, Claudia A; Alonso-Ferrero, Maria E; Blundell, Michael P; Schambach, Axel; Baum, Christopher; Malik, Punam; Thrasher, Adrian J; Jordan, Michael B; Gaspar, H Bobby (2015. ) Perforin gene transfer into hematopoietic stem cells improves immune dysregulation in murine models of perforin deficiency.Molecular therapy : the journal of the American Society of Gene Therapy, , 23 (4 ) ,737-45 More Information

Li, Chen; Gonsalves, Caryn S; Eiymo Mwa Mpollo, Marthe-Sandrine; Malik, Punam; Tahara, Stanley M; Kalra, Vijay K (2015. ) MicroRNA 648 Targets ET-1 mRNA and is cotranscriptionally regulated with MICAL3 by PAX5.Molecular and cellular biology, , 35 (3 ) ,514-28 More Information

Chang, Kyung Hee; Nayak, Ramesh C; Roy, Swarnava; Perumbeti, Ajay; Wellendorf, Ashley M; Bezold, Katie Y; Pirman, Megan; Hill, Sarah E; Starnes, Joseph; Loberg, Anastacia; Zhou, Xuan; Inagami, Tadashi; Zheng, Yi; Malik, Punam; Cancelas, Jose A (2015. ) Vasculopathy-associated hyperangiotensinemia mobilizes haematopoietic stem cells/progenitors through endothelial AT?R and cytoskeletal dysregulation.Nature communications, , 6 ,5914 More Information

Li, Chen; Mpollo, Marthe-Sandrine Eiymo Mwa; Gonsalves, Caryn S; Tahara, Stanley M; Malik, Punam; Kalra, Vijay K (2014. ) Peroxisome proliferator-activated receptor-?-mediated transcription of miR-199a2 attenuates endothelin-1 expression via hypoxia-inducible factor-1?.The Journal of biological chemistry, , 289 (52 ) ,36031-47 More Information

Suzuki, Takuji; Arumugam, Paritha; Sakagami, Takuro; Lachmann, Nico; Chalk, Claudia; Sallese, Anthony; Abe, Shuichi; Trapnell, Cole; Carey, Brenna; Moritz, Thomas; Malik, Punam; Lutzko, Carolyn; Wood, Robert E; Trapnell, Bruce C (2014. ) Pulmonary macrophage transplantation therapy.Nature, , 514 (7523 ) ,450-4 More Information

Chandrakasan, Shanmuganathan; Malik, Punam (2014. ) Gene therapy for hemoglobinopathies: the state of the field and the future.Hematology/oncology clinics of North America, , 28 (2 ) ,199-216 More Information

Kiem, Hans-Peter; Arumugam, Paritha I; Burtner, Christopher R; Fox, Catherine F; Beard, Brian C; Dexheimer, Phillip; Adair, Jennifer E; Malik, Punam (2014. ) Pigtailed macaques as a model to study long-term safety of lentivirus vector-mediated gene therapy for hemoglobinopathies.Molecular therapy. Methods & clinical development, , 1 ,14055 More Information

Zhou, Xuan; Florian, Maria Carolina; Arumugam, Paritha; Chen, Xiaoyi; Cancelas, Jose A; Lang, Richard; Malik, Punam; Geiger, Hartmut; Zheng, Yi (2013. ) RhoA GTPase controls cytokinesis and programmed necrosis of hematopoietic progenitors.The Journal of experimental medicine, , 210 (11 ) ,2371-85 More Information

punam.malik@cchmc.org