scholarly journals MicroRNA‐based recombinant AAV vector assembly improves efficiency of suicide gene transfer in a murine model of lymphoma

2020 ◽  
Vol 9 (9) ◽  
pp. 3188-3201 ◽  
Author(s):  
Nusrat Khan ◽  
Sabna Cheemadan ◽  
Himanshi Saxena ◽  
Sridhar Bammidi ◽  
Giridhara R. Jayandharan
Keyword(s):  
Gene Transfer ◽  
Murine Model ◽  
Suicide Gene ◽  
Aav Vector

scholarly journals Thymidine phosphorylase is both a therapeutic and a suicide gene in a murine model of mitochondrial neurogastrointestinal encephalomyopathy

Gene Therapy ◽  
2014 ◽  
Vol 21 (7) ◽  
pp. 673-681 ◽  
Author(s):  
S López-Estévez ◽  
G Ferrer ◽  
J Torres-Torronteras ◽  
M J Mansilla ◽  
S Casacuberta-Serra ◽  
...  
Keyword(s):  
Murine Model ◽  
Thymidine Phosphorylase ◽  
Suicide Gene ◽  
Mitochondrial Neurogastrointestinal Encephalomyopathy

Systemic AAV9.LAMP2B injection reverses metabolic and physiologic multiorgan dysfunction in a murine model of Danon disease

2020 ◽  
Vol 12 (535) ◽  
pp. eaax1744 ◽  
Author(s):  
Ana Maria Manso ◽  
Sherin I. Hashem ◽  
Bradley C. Nelson ◽  
Emily Gault ◽  
Angel Soto-Hermida ◽  
...  
Keyword(s):  
Skeletal Muscle ◽  
Gene Transfer ◽  
Murine Model ◽  
Autophagic Flux ◽  
Adeno Associated Virus ◽  
Multiorgan Dysfunction ◽  
Danon Disease ◽  
Physiologic Function ◽  
Male Patients ◽  
Dose Dependent

Danon disease (DD) is a rare X-linked autophagic vacuolar myopathy associated with multiorgan dysfunction, including the heart, skeletal muscle, and liver. There are no specific treatments, and most male patients die from advanced heart failure during the second or third decade of life. DD is caused by mutations in the lysosomal-associated membrane protein 2 (LAMP2) gene, a key mediator of autophagy. LAMP2 has three isoforms: LAMP2A, LAMP2B, and LAMP2C. LAMP2B is the predominant isoform expressed in cardiomyocytes. This study evaluates the efficacy of human LAMP2B gene transfer using a recombinant adeno-associated virus 9 carrying human LAMP2B (AAV9.LAMP2B) in a Lamp2 knockout (KO) mouse, a DD model. AAV9.LAMP2B was intravenously injected into 2- and 6-month-old Lamp2 KO male mice to assess efficacy in adolescent and adult phenotypes. Lamp2 KO mice receiving AAV9.LAMP2B demonstrated dose-dependent restoration of human LAMP2B protein in the heart, liver, and skeletal muscle tissue. Impaired autophagic flux, evidenced by increased LC3-II, was abrogated by LAMP2B gene transfer in all tissues in both cohorts. Cardiac function was also improved, and transaminases were reduced in AAV9.LAMP2B-treated KO mice, indicating favorable effects on the heart and liver. Survival was also higher in the older cohort receiving high vector doses. No anti-LAMP2 antibodies were detected in mice that received AAV9.LAMP2B. In summary, LAMP2B gene transfer improves metabolic and physiologic function in a DD murine model, suggesting that a similar therapeutic approach may be effective for treating patients with this highly morbid disease.

scholarly journals 759. Reduction of HLH-like Manifestations in Murine Model of Munc13-4 Deficiency Following Lentiviral Gene Transfer into Hematopoietic Stem and Progenitor Cells

2016 ◽  
Vol 24 ◽  
pp. S300
Author(s):  
Tayebeh-Shabi Soheili ◽  
Fernando Sepulveda ◽  
Amandine Durand ◽  
Julie Rivière ◽  
Samia Martin ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Progenitor Cells ◽  
Murine Model ◽  
Hematopoietic Stem ◽  
Stem And Progenitor Cells

Imaging expression of adenoviral HSV1-tk suicide gene transfer using the nucleoside analogue FIRU

2002 ◽  
Vol 29 (7) ◽  
pp. 939-947 ◽  
Author(s):  
Dharmin Nanda ◽  
Marion de Jong ◽  
Ronald Vogels ◽  
Menzo Havenga ◽  
Maarten Driesse ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Nucleoside Analogue ◽  
Suicide Gene

scholarly journals 730. T Cell Responses to AAV Vector Capsid in Normal Donors and Subjects Who Have Undergone Liver-Directed AAV-Mediated Gene Transfer

2006 ◽  
Vol 13 ◽  
pp. S282 ◽  
Author(s):  
Marcela V. Maus ◽  
Federico Mingozzi ◽  
Denise E. Sabatino ◽  
Daniel Hui ◽  
Margaret V. Ragni ◽  
...  
Keyword(s):  
T Cell ◽  
Gene Transfer ◽  
T Cell Responses ◽  
Aav Vector ◽  
Cell Responses

scholarly journals AAV6 Vexosomes Mediate Robust Suicide Gene Delivery in a Murine Model of Hepatocellular Carcinoma

2020 ◽  
Vol 17 ◽  
pp. 497-504 ◽  
Author(s):  
Nusrat Khan ◽  
Shubham Maurya ◽  
Sridhar Bammidi ◽  
Giridhara R. Jayandharan
Keyword(s):  
Hepatocellular Carcinoma ◽  
Gene Delivery ◽  
Murine Model ◽  
Suicide Gene

scholarly journals Gene Therapy Using a Liver-targeted AAV Vector Restores Nucleoside and Nucleotide Homeostasis in a Murine Model of MNGIE

2014 ◽  
Vol 22 (5) ◽  
pp. 901-907 ◽  
Author(s):  
Javier Torres-Torronteras ◽  
Carlo Viscomi ◽  
Raquel Cabrera-Pérez ◽  
Yolanda Cámara ◽  
Ivano Di Meo ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Aav Vector

The influence of AAV2-mediated gene transfer of human IL-10 on neurodegeneration and immune response in a murine model of Parkinson's disease

2014 ◽  
Vol 66 (4) ◽  
pp. 660-669 ◽  
Author(s):  
Ilona Joniec-Maciejak ◽  
Agnieszka Ciesielska ◽  
Adriana Wawer ◽  
Anna Sznejder-Pachołek ◽  
Joanna Schwenkgrub ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Immune Response ◽  
Parkinson's Disease ◽  
Gene Transfer ◽  
Murine Model
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