scholarly journals Lentivirus-meditated frataxin gene delivery reverses genome instability in Friedreich ataxia patient and mouse model fibroblasts

Gene Therapy ◽  
2016 ◽  
Vol 23 (12) ◽  
pp. 846-856 ◽  
Author(s):  
H Khonsari ◽  
M Schneider ◽  
S Al-Mahdawi ◽  
Y G Chianea ◽  
M Themis ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Mouse Model ◽  
Genome Instability ◽  
Friedreich Ataxia ◽  
Frataxin Gene

scholarly journals HDAC Inhibitors Correct Frataxin Deficiency in a Friedreich Ataxia Mouse Model

PLoS ONE ◽  
2008 ◽  
Vol 3 (4) ◽  
pp. e1958 ◽  
Author(s):  
Myriam Rai ◽  
Elisabetta Soragni ◽  
Kai Jenssen ◽  
Ryan Burnett ◽  
David Herman ◽  
...  
Keyword(s):  
Mouse Model ◽  
Friedreich Ataxia ◽  
Hdac Inhibitors

scholarly journals Therapeutic potential of AAV9-S15D-RLC gene delivery in humanized MYL2 mouse model of HCM

2019 ◽  
Vol 97 (7) ◽  
pp. 1033-1047 ◽  
Author(s):  
Sunil Yadav ◽  
Chen-Ching Yuan ◽  
Katarzyna Kazmierczak ◽  
Jingsheng Liang ◽  
Wenrui Huang ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Mouse Model ◽  
Therapeutic Potential

scholarly journals Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX

Gene Therapy ◽  
2005 ◽  
Vol 13 (2) ◽  
pp. 117-126 ◽  
Author(s):  
B W Bigger ◽  
E K Siapati ◽  
A Mistry ◽  
S N Waddington ◽  
M S Nivsarkar ◽  
...  
Keyword(s):  
Stem Cell ◽  
Gene Delivery ◽  
Mouse Model ◽  
Human Factor ◽  
Factor Ix ◽  
Hemophilia B ◽  
Human Factor Ix ◽  
Cell Gene ◽  
Stem Cell Gene

Frataxin gene point mutations in Italian Friedreich ataxia patients

Neurogenetics ◽  
2007 ◽  
Vol 8 (4) ◽  
pp. 289-299 ◽  
Author(s):  
Cinzia Gellera ◽  
Barbara Castellotti ◽  
Caterina Mariotti ◽  
Rossana Mineri ◽  
Viviana Seveso ◽  
...  
Keyword(s):  
Friedreich Ataxia ◽  
Point Mutations ◽  
Frataxin Gene

RCAS-mediated Retroviral Gene Delivery - A Versatile Tool to Study Gene Function in a Mouse Model of Pancreatic Cancer

2008 ◽  
Vol 0 (ja) ◽  
pp. 081015093227032
Author(s):  
Ulrich Mayr ◽  
Alexander von Werder ◽  
Barbara Seidler ◽  
Wolfgang Reindl ◽  
Monther Bajbouj ◽  
...  
Keyword(s):  
Pancreatic Cancer ◽  
Gene Delivery ◽  
Mouse Model ◽  
Gene Function ◽  
Versatile Tool
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