Malik, Punam

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-Bey-Abina, S., Pai, S.-Y., Gaspar, H. B., Armant, M., Berry, C. C., Blanche, S., Bleesing, J., Blondeau, J., de Boer, H., Buckland, K. F., et al. (2014) · New England Journal of Medicine

Virus-based gene therapy researchImmunodeficiency and Autoimmune Disorders

Cited by 412

Cardiomyopathy With Restrictive Physiology in Sickle Cell Disease

Niss, O., Quinn, C. T., Lane, A., Daily, J., Khoury, P. R., Bakeer, N., Kimball, T. R., Towbin, J. A., Malik, P., & Taylor, M. D. (2016) · JACC: Cardiovascular Imaging

Hemoglobinopathies and Related DisordersCardiovascular Function and Risk Factors

Cited by 142

Association between diffuse myocardial fibrosis and diastolic dysfunction in sickle cell anemia

Niss, O., Fleck, R., Makue, F., Alsaied, T., Desai, P., Towbin, J. A., Malik, P., Taylor, M. D., & Quinn, C. T. (2017) · Blood

Hemoglobinopathies and Related DisordersCardiovascular Function and Risk Factors

Cited by 115

Robust clinical and laboratory response to hydroxyurea using pharmacokinetically guided dosing for young children with sickle cell anemia

McGann, P. T., Niss, O., Dong, M., Marahatta, A., Howard, T. A., Mizuno, T., Lane, A., Kalfa, T. A., Malik, P., Quinn, C. T., et al. (2019) · American Journal of Hematology

Hemoglobinopathies and Related DisordersPharmaceutical studies and practices

Cited by 105

The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors

Higashimoto, T., Urbinati, F., Perumbeti, A., Jiang, G., Zarzuela, A., Chang, L.-J., Kohn, D. B., & Malik, P. (2007) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 101

Molecular, phenotypic, and sample-associated data to describe pluripotent stem cell lines and derivatives

Daily, K., Ho Sui, S. J., Schriml, L. M., Dexheimer, P. J., Salomonis, N., Schroll, R., Bush, S., Keddache, M., Mayhew, C., Lotia, S., et al. (2017) · Scientific Data

Pluripotent Stem Cells ResearchDevelopmental Biology and Gene Regulation

Cited by 81

Sickle cell anemia mice develop a unique cardiomyopathy with restrictive physiology

Bakeer, N., James, J., Roy, S., Wansapura, J., Shanmukhappa, S. K., Lorenz, J. N., Osinska, H., Backer, K., Huby, A.-C., Shrestha, A., et al. (2016) · Proceedings of the National Academy of Sciences

Hemoglobinopathies and Related DisordersCardiomyopathy and Myosin Studies

Cited by 79

Scale-up and manufacturing of clinical-grade self-inactivating γ-retroviral vectors by transient transfection

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., et al. (2012) · Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 34