Concepedia

In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model

Chang Li, Hongjie Wang, Aphrodite Georgakopoulou, Sucheol Gil, Evangelia Yannaki, André Lieber

Molecular Therapy · 2020 · 70 citations · 51 references

DOIFull text

Open access

Concepts

References

51

Fast gapped-read alignment with Bowtie 2

Ben Langmead, Steven L. Salzberg · Nature Methods · 2012 · 61.1K citations

, , +11

BEDTools: a flexible suite of utilities for comparing genomic features

Aaron R. Quinlan, Ira M. Hall · Bioinformatics · 2010 · 30.4K citations · Full text

, , +14

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

Steven E. Raper, Narendra Chirmule, Frank S. Lee et al. · Molecular Genetics and Metabolism · 2003 · 1.5K citations

, , +14

An improved zinc-finger nuclease architecture for highly specific genome editing

Jeffrey C. Miller, Michael C. Holmes, Jianbin Wang et al. · Nature Biotechnology · 2007 · 1K citations

, , +13

Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates

Lajos Mátés, Marinee Chuah, Eyayu Belay et al. · Nature Genetics · 2009 · 997 citations

, , +12