AAV9 gene therapy

2 papers3 findings

1 lab in 1 country

Funders: Amicus Therapeutics and 3 more
3 of 3 findings with company ties

Animals1

Outcomes

3

Behavioral hallmarks of the disease

downin mice1

1 study

AAV9 gene therapy decreases behavioral hallmarks of the disease in mice.
“reducing histopathological and behavioral hallmarks of the disease”
Animalsmouse model of CLN8 diseasescAAV9-human CLN8
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Molecular therapy : the journal of the American Society of Gene Therapy24 Sep 2020

Histopathological hallmarks of CLN8 disease

downin mice1

1 study

AAV9 gene therapy decreases histopathological hallmarks of cln8 disease in mice.
“reducing histopathological and behavioral hallmarks of the disease”
Animalsmouse model of CLN8 diseasescAAV9-human CLN8
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Molecular therapy : the journal of the American Society of Gene Therapy24 Sep 2020

Lifespan

upin mice1

1 study

AAV9 gene therapy extends lifespan in mice (from 10 months to beyond 24 months).
“restoring lifespan from 10 months in untreated animals to beyond 24 months of age in treated animals”
Animalsmouse model of CLN8 diseasescAAV9-human CLN8
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease
Molecular therapy : the journal of the American Society of Gene Therapy24 Sep 2020

Latest

1 in 12 months

International Journal of Molecular SciencesYesterday

AAV9 Gene Therapy for Ovarian Dysfunction and Reproductive Aging: Molecular Mechanisms, Therapeutic Targets, and Translational Perspectives

ReviewReproductive system

Molecular Therapy24 Sep

AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

Experiments in miceInterventions