Published in
American Association for the Advancement of Science, Science Advances, 2(7), 2021
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Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberin
Journal article published in 2021 by
Pike-See Cheah 
,
Shilpa Prabhakar ,
David Yellen ,
Roberta L. Beauchamp,
Xuan Zhang,
Shingo Kasamatsu,
Roderick T. Bronson,
Elizabeth A. Thiele,
David J. Kwiatkowski ,
Anat Stemmer-Rachamimov ,
Bence György,
King-Hwa Ling ,
Masao Kaneki,
Bakhos A. Tannous ,
Vijaya Ramesh
and other authors.
,
Shilpa Prabhakar ,
David Yellen ,
Roberta L. Beauchamp,
Xuan Zhang,
Shingo Kasamatsu,
Roderick T. Bronson,
Elizabeth A. Thiele,
David J. Kwiatkowski ,
Anat Stemmer-Rachamimov ,
Bence György,
King-Hwa Ling ,
Masao Kaneki,
Bakhos A. Tannous ,
Vijaya Ramesh
and other authors.
This paper is made freely available by the publisher.
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Abstract
Gene therapy for tuberous sclerosis type 2 proved beneficial in a mouse model of the disease, extending life span.