Profile
My laboratory is interested in developing innovative therapeutic genome editing tools to treat patients with inherited hematological disorders such as sickle cell diseases, thalassemia and Hemophilia A&B. We are planning to directly manipulate the genome of human hematopoietic stem cells (HSCs) in a sequence specific manner using targeted genome engineering platform based on CRISPR/CAS9 system for the treatment of inherited hematological disorders. We are further working on the delivery platforms to increase the genome editing efficiency and reduce the off-target effects. We anticipate that our strategy will overcome the therapeutic barriers that currently limit clinical applications.
Current Focus Areas
My Current research is focused on developing preclinical genome editing strategies for the treatment of patients with inherited hematological disorders such as sickle cell diseases, beta thalassemia and hemophilia
Selected Publications
1. Vignesh Rajendiran*, Nivedhitha Devaraju*, Nithin Sam Ravi, Lokesh Panigrahi, Joshua Paul, Chandrasekar Gopalakrishnan, Stacia Wyman, Keerthiga Ariudainambi, Gokulnath Mahalingam, Yogapriya Periyasami, Kirti Prasad, Anila George, Dhiyaneshwaran Sukumaran, Sandhiya Gopinathan, Aswin Anand Pai,Yukio Nakamura, Poonkuzhali Balasubramanian, Rajasekaran Ramalingam, Saravanabhavan Thangavel, Shaji R Velayudhan, Jacon E Corn, Merlin Crossley, Srujan Marepally, Alok Srivastava, Mohankumar KM (2024) Base editing of key residues in the BCL11A-XL-specific zinc finger domains de-represses fetal globin expression. Mol Therapy 32(3):663-677
2. Ravi NS, Wienert B, Wyman SK, Bell H, Vu J, Pai AA, Balasubramanian P, Nakamura Y, Kurita R, Marepally S, Thangavel S, Shaji RV, Srivastava A, DeWitt MA, Crossley M, Corn JE, Mohankumar KM (2022). Identification of novel HPFH-like mutations by CRISPR base editing that elevates the expression of fetal hemoglobin. eLife.11;11: e65421.
3. George A, Ravi NS, Prasad K, Panigrahi L, Koikkara S, Rajendiran V, Devaraju N, Paul J, Pai AA, Nakamura Y, Kurita R, Balasubramanian P, Thangavel S, Marepally S, Velayudhan SR, Srivastava A and Mohankumar KM (2022) Efficient and error-free correction of sickle mutation in human erythroid cells using prime editor-2. Front. Genome Ed. 4:1085111
4. Mohankumar KM, Currle DS, White E, Boulos N, Dapper J, Eden C, Nimmervoll B, Thiruvenkatam R, Connelly M, Kranenburg TA, Neale G, Olsen S, Wang M, Finkelstein D, Wright K, Gupta K, Ellison DW, Thomas AO, Gilbertson RJ. In vivo oncogenomics screen identifies novel ependymoma oncogenes and tumor suppressors within large chromosomal alterations. Nature Genetics. 47:878-87
5. Matthew Parker*, Mohankumar KM*, Punchihewa C *, Weinlich R*, Dalton JD, Lee R, Ruth G, Tatevossian RG, Phoenix TN, Thiruvenkatam R, Li Y, White E, Tang B, Orisme W, Gupta K, Rusch M, Chen X, Nagahawhatte P, Hedlund E, Finkelstein D, Wu G, Shurtleff S, Easton J, Boggs K, Yergeau D, VadodariaB, Mulder HL, Becksford J, Gupta P, Huether R, Ma J, Song G, Ding L, Lu C, Ochoa K, Zhao D, Fulton RS, Fulton LL, Mardis ER, Wilson RK, Downing JR, Green D, Zhang J, Ellison DW, and Gilbertson RJ (2014) Highly recurrent C11ORF95-RELA fusions drive oncogenic NF-κB signaling in Ependymoma. Nature. 506:451-455