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CRISPR-Cas9 Technology for Combating Genetic Disorders
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Author(s): Ajay Sharma (Lovely Professional University, India)and Pratibha Singh (Lovely Professional University, India)
Copyright: 2026
Pages: 28
Source title:
CRISPR-Cas9 Technology for Precision Medicine
Source Author(s)/Editor(s): Jen-Tsung Chen (National University of Kaohsiung, Taiwan)
DOI: 10.4018/979-8-3373-4857-5.ch005
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Abstract
The CRISPR-Cas9 technique, which offers an unparalleled degree of precision and efficacy in genome editing, has entirely changed the area of genetic disorders medicine. Since its discovery as a component of the bacterial immune defence system, CRISPR-Cas9 has quickly been modified for use in therapeutic use, most notably in the fight against genetic disease problems. The essential mechanics of CRISPR-Cas9, along with its innovative potential in the treatment of complicated and monogenic genetic illnesses, are discussed in the manuscript. The author has gained insights into effective preclinical and clinical uses, such as the management of sickle cell anaemia, Duchenne muscular dystrophy, and cystic fibrosis, as well as new developments like base editing and prime editing. This chapter also sheds light on important topics such as off-target effects, delivery system constraints, ethical impasses, and regulatory apprehensions. Through a combination of case studies and recent developments, this chapter attempts to give a detailed overview of how CRISPR-Cas9 is changing precision medicine and developing new treatment paradigms for genetic disorders that were previously incurable.
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