The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix.The ability to modify selectively specific genes ...The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix.The ability to modify selectively specific genes provides a powerful tool for characterizing gene functions,performing gene therapy,correcting specific genetic mutations,eradicating diseases,engineering cells and organisms to achieve new and different functions and obtaining transgenic animals as models for studying specific diseases.Clustered regularly interspaced short palindromic repeats(CRISPR)/Cas9 technology has recently revolutionized genome engineering.The application of this new technology to stem cell research allows disease models to be developed to explore new therapeutic tools.The possibility of translating new systems of molecular knowledge to clinical research is particularly appealing for addressing degenerative diseases.In this review,we describe several applications of CRISPR/Cas9 to stem cells related to degenerative diseases.In addition,we address the challenges and future perspectives regarding the use of CRISPR/Cas9 as an important technology in the medical sciences.展开更多
文摘The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix.The ability to modify selectively specific genes provides a powerful tool for characterizing gene functions,performing gene therapy,correcting specific genetic mutations,eradicating diseases,engineering cells and organisms to achieve new and different functions and obtaining transgenic animals as models for studying specific diseases.Clustered regularly interspaced short palindromic repeats(CRISPR)/Cas9 technology has recently revolutionized genome engineering.The application of this new technology to stem cell research allows disease models to be developed to explore new therapeutic tools.The possibility of translating new systems of molecular knowledge to clinical research is particularly appealing for addressing degenerative diseases.In this review,we describe several applications of CRISPR/Cas9 to stem cells related to degenerative diseases.In addition,we address the challenges and future perspectives regarding the use of CRISPR/Cas9 as an important technology in the medical sciences.
