发明名称 Transfection, storage and transfer of male germ cells for generation of transgenic species and genetic therapies
摘要 A composition for in vitro and in vivo transfection of vertebrate male germ cells comprises a nucleic acid or transgene, and a gene delivery system, and optionally a protective internalizing agent, such as an endosomal lytic agent, a virus or a viral component, which is internalized by cells along with the transgene and which enhances gene transfer through the cytoplasm to the nucleus of the male germ cell. A method of genetically altering a vertebrate male germ cell in vivo employs a lentiviral-derived vector. A method of substantially depopulating a vertebrate testis employs a combination of a dose of an alkylating agent, such as busulfan, chlorambucil, cyclophosphamide, melphalan, or ethyl ethanesulfonic acid, and a dose of gamma radiation. A pharmaceutical preparation and a transfer kit utilize the composition. A method for introducing a polynucleotide into vertebrate male germ cells comprises the administration of the composition to a vertebrate. A method for isolating or selecting transfected cells utilizes a reporter gene, and a method for administering transfected male germ cells utilizes male germ cells which have been transfected in vitro.
申请公布号 US6734338(B1) 申请公布日期 2004.05.11
申请号 US19990311599 申请日期 1999.05.13
申请人 CEDARS-SINAI MEDICAL CENTER;IMPEL, IMPERIAL COLLEGE OF SCIENCE, TECHNOLOGY AND MEDICINE 发明人 READHEAD CAROL W.;WINSTON ROBERT
分类号 A01K67/027;A61K35/12;A61K48/00;C12N5/076;(IPC1-7):C12P21/00;C12H15/00 主分类号 A01K67/027
代理机构 代理人
主权项
地址