CRISPR/Cas9 Editing: Sparking Discussion on Safety in Light of the Need for New Therapeutics.
Hum Gene Ther
; 31(15-16): 794-807, 2020 08.
Article
em En
| MEDLINE
| ID: mdl-32586150
ABSTRACT
Recent advances in genome sequencing have greatly improved our ability to understand and identify the causes of genetic diseases. However, there remains an urgent need for innovative, safe, and effective treatments for these diseases. CRISPR-based genome editing systems have become important and powerful tools in the laboratory, and efforts are underway to translate these into patient therapies. Therapeutic base editing is one form of genome engineering that has gained much interest because of its simplicity, specificity, and effectiveness. Base editors are a fusion of a partially deactivated Cas9 enzyme with nickase function, together with a base-modifying enzyme. They are capable of precisely targeting and repairing a pathogenic mutation to restore the normal function of a gene, ideally without disturbing the rest of the genome. In the past year, research has identified new safety concerns of base editors and sparked new innovations to improve their safety. In this review, we provide an overview of the recent advances in the safety and effectiveness of therapeutic base editors and prime editing.
Palavras-chave
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
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Genoma Humano
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Sistemas CRISPR-Cas
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Edição de Genes
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Mutação
Limite:
Humans
Idioma:
En
Revista:
Hum Gene Ther
Assunto da revista:
GENETICA MEDICA
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TERAPEUTICA
Ano de publicação:
2020
Tipo de documento:
Article