
CRISPR editing of HPFH3 genotype induces γ-globin expression and reverses sickle cell disease and β-thalassemia phenotypes
Institute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025 India · Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002 India · Laboratory of Nanotechnology and Chemical Biology, Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3Rd Milestone, Faridabad-Gurgaon Expressway, Faridabad, Haryana 121001 India · Thalassemia and Sickle Cell Society- Kamala Hospital and Research Centre, Shivarampally, Hyderabad, 5000052 India · Rajiv Gandhi Cancer Institute and Research Institute and Research Centre, Rohini, Delhi, 110085 India · Cell Engineering Division, RIKEN BioResource, Research Center, Ibaraki, 3050074 Japan · Department of Biological Sciences and Bioengineering and Mehta Family Centre for Engineering in Medicine, Indian Institute of Technology Kanpur, Kanpur, UP 208016 IndiaInstitute for Genomics and Integrative Biology, Mathura Road, Sukhdev Vihar, New Delhi, 110025 India
- Academy of Scientific and Innovative Research (AcSIR), Ghaziabad, 201002 India
- Laboratory of Nanotechnology and Chemical Biology, Regional Centre for Biotechnology, NCR Biotech Science Cluster, 3Rd Milestone, Faridabad-Gurgaon Expressway, Faridabad, Haryana 121001 India
- Thalassemia and Sickle Cell Society- Kamala Hospital and Research Centre, Shivarampally, Hyderabad, 5000052 India
- Rajiv Gandhi Cancer Institute and Research Institute and Research Centre, Rohini, Delhi, 110085 India
- Cell Engineering Division, RIKEN BioResource, Research Center, Ibaraki, 3050074 Japan
- Department of Biological Sciences and Bioengineering and Mehta Family Centre for Engineering in Medicine, Indian Institute of Technology Kanpur, Kanpur, UP 208016 India
Summary: Researchers use CRISPR to recreate a naturally protective genetic pattern that increases fetal haemoglobin. Experiments in cells and disease models investigate whether this approach could counter sickle cell disease and beta-thalassaemia. It is a preclinical study, not proof of a new approved treatment.
