CRISPR/CAS9-BASED COMPOSITIONS AND METHODS FOR TREATING CANCER

• Main Authors: ZACK DONALD, BUNZ FRED, JASKULA-RANGA VINOD
• Format: Patent
• Language: Chinese; English
• Published: 02.07.2019
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

Described herein are methods for preventing, inhibiting, or treating cancer in a subject. Also provided herein are methods of altering expression of one or more gene products in a cell, such as a cancer cell. Such methods may comprise utilizing a modified nuclease system, such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR associated (Cas) 9 (CRISPR-Cas9) comprising abidirectional HI promoter and gRNAs directed to oncogenes (rAAV-Onco-CRISPR) or tumor suppressor genes (rAAV-TSG) packaged in a compact adeno-associated virus (AAV) particle. Such methods may comprise co-administering or concurrently providing a recombinant adeno-associated virus-packaging adenovirus (Ad-rAAVpack) with the nuclease system. 本文描述用于预防、抑制或治疗受试者中的癌症的方法。本文还提供改变细胞，例如癌细胞中的一种或多种基因产物的表达的方法。这种方法可以包括利用修饰的核酸酶系统，例如成簇规则间隔短回文重复(CRISPR)/CRISPR相关(Cas)9(CRISPR-Cas9)，其包含双向H1启动子和针对包装在致密的腺相关病毒(AAV)颗粒中的癌基因(rAAV-Onco-CRISPR)或肿瘤抑制基因(rAAV-TSG)的gRNA。这种方法可以包括与核酸酶系统共同施用或同时提供重组腺相关病毒包装腺病毒(Ad-rAAVpack)。