CRISPR-based gene therapies have shown significant promise in treating inherited disorders, particularly in blood and neuromuscular diseases. This review discusses the development and clinical applications of CRISPR/Cas9 technology in these areas. In blood disorders, CRISPR/Cas9 has been used to correct mutations in the β-globin gene, leading to the first FDA-approved CRISPR-based therapy for sickle cell disease and β-thalassemia. The therapy involves editing hematopoietic stem cells to increase fetal hemoglobin production, reducing the need for blood transfusions. Similar approaches are being explored for other blood disorders, including primary immunodeficiencies. In neuromuscular diseases, CRISPR/Cas9 has been used to generate models of Duchenne muscular dystrophy (DMD) and to develop therapeutic strategies aimed at restoring dystrophin expression. Various approaches, including exon deletion, skipping, and reframing, have been tested in preclinical models, with some showing promising results. However, challenges remain, including efficient in vivo delivery, safety concerns, and the need for long-term efficacy. The review highlights the potential of CRISPR-based therapies in treating DMD and other genetic disorders, emphasizing the need for further research and clinical trials to address current limitations and improve therapeutic outcomes.CRISPR-based gene therapies have shown significant promise in treating inherited disorders, particularly in blood and neuromuscular diseases. This review discusses the development and clinical applications of CRISPR/Cas9 technology in these areas. In blood disorders, CRISPR/Cas9 has been used to correct mutations in the β-globin gene, leading to the first FDA-approved CRISPR-based therapy for sickle cell disease and β-thalassemia. The therapy involves editing hematopoietic stem cells to increase fetal hemoglobin production, reducing the need for blood transfusions. Similar approaches are being explored for other blood disorders, including primary immunodeficiencies. In neuromuscular diseases, CRISPR/Cas9 has been used to generate models of Duchenne muscular dystrophy (DMD) and to develop therapeutic strategies aimed at restoring dystrophin expression. Various approaches, including exon deletion, skipping, and reframing, have been tested in preclinical models, with some showing promising results. However, challenges remain, including efficient in vivo delivery, safety concerns, and the need for long-term efficacy. The review highlights the potential of CRISPR-based therapies in treating DMD and other genetic disorders, emphasizing the need for further research and clinical trials to address current limitations and improve therapeutic outcomes.