Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

The development of the next-generation gene therapy vectors for hemophilia requires using lower and thus potentially safer vector doses and augmenting their therapeutic efficacy. We have identified hepatocyte-specific transcriptional cis-regulatory modules (CRMs) by using a computational strategy th...

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Detalhes bibliográficos
Main Authors: Nair, Nisha, Rincon, Melvin Y., Evens, Hanneke, Sarcar, Shilpita, Dastidar, Sumitava, Samara-Kuko, Emira, Ghandeharian, Omid, Man Viecelli, Hiu, Thöny, Beat, De Bleser, Pieter, VandenDriessche, Thierry, Chuah, Marinee K.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2014
Assuntos:
Thrombosis and Hemostasis
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4023424/
https://ncbi.nlm.nih.gov/pubmed/24637359
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2013-10-534032
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