H. Fang
发表
First-in-human case report: AAV9-hGAA gene therapy for a patient with infantile-onset Pompe disease.
Z. Lina,
X. Hui,
L. Jun,
2022,
medRxiv.
S. Levy,
T. Hurd,
David S. Williams,
2014,
American journal of human genetics.
T. Chan,
R. Lifton,
D. Milford,
2016,
Nature Communications.
F. Hildebrandt,
S. Ashraf,
V. Vega-Warner,
2014,
Clinical journal of the American Society of Nephrology : CJASN.
S. Levy,
T. Hurd,
O. Gribouval,
2013,
The Journal of clinical investigation.
Whole exome resequencing distinguishes cystic kidney diseases from phenocopies in renal ciliopathies
S. Levy,
N. Katsanis,
T. Hurd,
2013,
Kidney international.
A. Paterson,
H. Prokisch,
S. Levy,
2013,
The Journal of clinical investigation.
S. Engelmann,
R. Lifton,
F. Hildebrandt,
2015,
Journal of the American Society of Nephrology : JASN.
Margaret Y. Nettleton,
S. Levy,
R. Lifton,
2015,
The Journal of clinical investigation.
S. Levy,
M. Simpson,
F. Hildebrandt,
2015,
American journal of human genetics.
Yan-Feng Zhang,
Jing-Yi Jin,
Yuzhong Xie,
2015
.
ADCK 4 mutations promote steroid-resistant nephrotic syndrome through CoQ 10 biosynthesis disruption
A. Paterson,
H. Prokisch,
S. Levy,
2018
.
Margaret Y. Nettleton,
S. Levy,
R. Lifton,
2015,
The Journal of clinical investigation.