Esrick, Erica B.

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Wu, Y., Zeng, J., Roscoe, B. P., Liu, P., Yao, Q., Lazzarotto, C. R., Clement, K., Cole, M. A., Luk, K., Baricordi, C., et al. (2019) · Nature Medicine

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 515

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

Esrick, E. B., Lehmann, L. E., Biffi, A., Achebe, M., Brendel, C., Ciuculescu, M. F., Daley, H., MacKinnon, B., Morris, E., Federico, A., et al. (2021) · New England Journal of Medicine

Hemoglobinopathies and Related DisordersVirus-based gene therapy research

Cited by 449

Correction of Sickle Cell Disease in Adult Mice by Interference with Fetal Hemoglobin Silencing

Xu, J., Peng, C., Sankaran, V. G., Shao, Z., Esrick, E. B., Chong, B. G., Ippolito, G. C., Fujiwara, Y., Ebert, B. L., Tucker, P. W., et al. (2011) · Science

Hemoglobinopathies and Related DisordersHemoglobin structure and function

Cited by 326

Preclinical evaluation of a novel lentiviral vector driving lineage-specific BCL11A knockdown for sickle cell gene therapy

Brendel, C., Negre, O., Rothe, M., Guda, S., Parsons, G., Harris, C., McGuinness, M., Abriss, D., Tsytsykova, A., Klatt, D., et al. (2020) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchHemoglobinopathies and Related Disorders

Cited by 72

Clonal selection of hematopoietic stem cells after gene therapy for sickle cell disease

Spencer Chapman, M., Cull, A. H., Ciuculescu, M. F., Esrick, E. B., Mitchell, E., Jung, H., O’Neill, L., Roberts, K., Fabre, M. A., Williams, N., et al. (2023) · Nature Medicine

Virus-based gene therapy researchHematopoietic Stem Cell Transplantation

Cited by 67