Vectores virales AAV para terapia génica contra el alcoholismo: Inhibición de la enzima aldehído deshidrogenasa mitocondrial en células de hepatoma humanas

AC Sánchez Daza - 2017 - repositorio.uchile.cl
El alcoholismo es un grave problema socio-económico. El costo asociado al abuso del
alcohol se ha estimado en 1.300 millones de dólares para la economía chilena y en 185 …

Gene Therapy for Bleeding Disorders

PE Monahan, YL Abajas - … Expert Clinical Review: Questions and Answers, 2016 - Springer
Gene therapy refers to any therapy in which the therapeutic entity is a nucleic acid. Gene
therapy most commonly delivers a DNA sequence, the transcription of which produces a …

Strategies for the Gene Modification of Megakaryopoiesis and Platelets

LJL Rey, U Modlich - Molecular and Cellular Biology of Platelet Formation …, 2016 - Springer
Platelets are the central players controlling blood hemostasis. Deregulation of their function
leads to various bleeding disorders in human patients with sometimes life-threatening …

Rational design for enhanced gene therapy with DNA transposons

PB Hackett, EL Aronovich - Molecular Therapy, 2014 - cell.com
© The American Society of Gene & Cell Therapy commentary the ribosome waits for a
charged transfer RNA. However, codon optimization in these studies seemingly had the …

[引用][C] Gene Therapy Advancements in Murine Phenylketonuria (PKU): A Dissertation

DY Richards - 2020 - Oregon Health & Science University

[引用][C] Blood First Edition Paper, prepublished online December 15, 2017; DOI 10.1182/blood-2017-09-804419

W Miesbach, K Meijer, M Coppens, P Kampmann… - 2017