CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes
Year: 2025
Type: article
Language: en
Topic: Hemoglobinopathies and Related Disorders
Authors and affiliations:
raw_author_name: Sangam Giri Goswami
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Pragya Gupta
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: V. R. Arvinden
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Nupur Bhargava
raw_affiliation_strings: CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Aditya Ramdas Iyer
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Vinodh Saravanakumar
raw_affiliation_strings: CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Poonam Yadav
raw_affiliation_strings: Laboratory of Nanotechnology and Chemical Biology, Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3Rd Milestone, Faridabad-Gurgaon Expressway, Faridabad, Haryana, 121001, India
raw_author_name: Somesh K. Jha
raw_affiliation_strings: Laboratory of Nanotechnology and Chemical Biology, Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3Rd Milestone, Faridabad-Gurgaon Expressway, Faridabad, Haryana, 121001, India
raw_author_name: Shivani Singh
raw_affiliation_strings: CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Ajay Kumar
raw_affiliation_strings: CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Praveen Singh
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India
raw_author_name: Padma Gunda
raw_affiliation_strings: Thalassemia and Sickle Cell Society- Kamala Hospital and Research Centre, Shivarampally, Hyderabad, 5000052, India
raw_author_name: Suman Jain
raw_affiliation_strings: Thalassemia and Sickle Cell Society- Kamala Hospital and Research Centre, Shivarampally, Hyderabad, 5000052, India
raw_author_name: Pallavi Mehta
raw_affiliation_strings: Rajiv Gandhi Cancer Institute and Research Institute and Research Centre, Rohini, Delhi, 110085, India
raw_author_name: Yukio Nakamura
raw_affiliation_strings: Cell Engineering Division, RIKEN BioResource, Research Center, Ibaraki, 3050074, Japan
raw_author_name: Ryo Kurita
raw_affiliation_strings: Cell Engineering Division, RIKEN BioResource, Research Center, Ibaraki, 3050074, Japan
raw_author_name: Avinash Bajaj
raw_affiliation_strings: Laboratory of Nanotechnology and Chemical Biology, Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3Rd Milestone, Faridabad-Gurgaon Expressway, Faridabad, Haryana, 121001, India
raw_author_name: Sivaprakash Ramalingam
raw_affiliation_strings: Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002, India. sivaprakash@iitk.ac.in
CSIR- Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025, India. sivaprakash@iitk.ac.in
Department of Biological Sciences and Bioengineering and Mehta Family Centre for Engineering in Medicine, Indian Institute of Technology Kanpur, Kanpur, UP, 208016, India. sivaprakash@iitk.ac.in