FEDERICO MINGOZZI,JUNWEI SUN,KATHERINE A. HIGH,PHILIP JOHNSON
申请号：
BR112014020325
公开号：
BR112014020325A2
申请日:
2013.02.19
申请国别(地区):
BR
年份:
2017
代理人:
摘要：
abstract the invention relates to adeno-associated virus (aav) serotype aav-rh74 and related aav vectors, and aav-rh74 and related aav vector mediated gene transfer methods and uses. in particular, aav-rh74 targets polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences. _________________________________________________________________ abstract translated abstract patent summary: "aav vector compositions and methods for gene transfer to cells, organs and tissues". The invention relates to aav-rh74 adeno-associated virus (aav) serotype and related aav vectors, and aav-rh74 and related aav vector mediated gene transfer methods and uses. in particular, aav-rh74 directs polynucleotides to cells, tissues or organs for the expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed in inhibiting nucleic acid sequences.abstract the invention relates to adeno-associated virus (aav) serotype aav-rh74 and related aav vectors, and aav-rh74 and related aav vector mediated gene transfer methods and uses. in particular, aav-rh74 targets polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences. _________________________________________________________________ resumo traduzido resumo patente de invenção: "composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos". a invenção refere-se ao sorotipo do vírus adeno-associado (aav) aav-rh74 e vetores do aav relacionados, e aav-rh74 e métodos de transferência do gene mediado pelo vetor do aav relacionado e utilizações. em particular, o aav-rh74 direciona os polinucleotídeos para as células, tec
