FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ;CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED, M.P.; Medioambientales y Tecnologicas;CENTRO DE INVESTIGACIONES ENERGETICAS, MEDIOAMBIENTALES Y TECNOLOGICAS;Centro de Investigaciones Energeticas
发明人:
Segovia, Jose C.,Gomez, Maria G.,Navarro, Susana,Meza, Nestor,Bueren, Juan,Bravo, Maria G.
申请号:
AU2017254665
公开号:
AU2017254665A1
申请日:
2017.04.20
申请国别(地区):
AU
年份:
2018
代理人:
摘要:
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.