Five years of successful inducible transgene expression following locoregional adeno-associated virus delivery in nonhuman primates with no detectable immunity

M Guilbaud, M Devaux, C Couzinie, J Le Duff… - Human Gene …, 2019 - liebertpub.com
long-term inducible expression up to at least 5 years post gene transfer, with no any detectable
host immune response against the transactivatorrecombinant adeno-associated virus

Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies

D Stone, M Aubert, KR Jerome - Gene Therapy, 2023 - nature.com
Recombinant AAV vectors have characteristics that make … These observations suggest that
additional long-term dose … such as the tetracycline-controlled transactivator (tTa) and reverse-…

Peripherally delivered Adeno-associated viral vectors for spinal cord injury repair

JD Sydney-Smith, AB Spejo, PM Warren… - Experimental …, 2022 - Elsevier
… can lead to long-term expression in nonhuman primates but in … Many transactivators are
not derived from mammals and … be rejected by the immune system in humans in the long term; …

A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo

G Zhong, H Wang, W He, Y Li, H Mou, ZJ Tickner… - Nature …, 2020 - nature.com
… of adeno-associated virus (AAV)-delivered transgenes, … transactivator protein for the Tet-On
transcriptional activation … of plasma Epo after the administration of recombinant protein 32,36 …

Timed GDNF gene therapy using an immune-evasive gene switch promotes long distance axon regeneration

R Eggers, F De Winter, SA Hoyng, RC Hoeben… - Brain, 2019 - academic.oup.com
… Here we used an immune-evasive transactivator generated … short- and sustained long-term
GDNF expression in vivo in … intraspinal adeno-associated viral vector-mediated expression of …

[HTML][HTML] Characterization of an immune-evading doxycycline-inducible lentiviral vector for gene therapy in the spinal cord

F De Winter, IF Quijorna, E Burnside, B Hobo… - Experimental …, 2022 - Elsevier
… -controlled transactivator) from the immune system we … In this study, we compared the
long-term performance of the … Adeno-associated viral vector-mediated neurotrophin gene …

[PDF][PDF] 5 years of successful inducible transgene expression following locoregional AAV delivery in nonhuman primates with no detectable immunity

M Guilbaud, M Devaux, C Couzinié, J Le Duff… - Human Gene …, 2019 - hal.science
recombinant Adeno-Associated Viruses (… long term inducible expression up to at least 5
years post-gene transfer with no any detectable host immune response against the transactivator

Control of mammalian gene expression by modulation of polyA signal cleavage at 5′ UTR

L Luo, JDY Jea, Y Wang, PW Chao, L Yen - Nature Biotechnology, 2024 - nature.com
… gene and cell therapy, such as adeno-associated virus (AAV), have intrinsic … ‘transactivator
protein can trigger immune responses in … in vivo in a tunable manner with long-term stability. …

[HTML][HTML] Externally-controlled systems for immunotherapy: from bench to bedside

M Tristán-Manzano, P Justicia-Lirio… - Frontiers in …, 2020 - frontiersin.org
… control transgene expression in the absence of transactivators in … in expression, long-term
and rapid decrease of the desired … of this strategy used two adeno-associated vectors (AAVs), …

[HTML][HTML] Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy

S Cheng, MM van Gaalen, M Bähr… - … Therapy-Methods & …, 2021 - cell.com
… the tetracycline-dependent transactivator correlates with long-term doxycycline-regulated
transgene expression in … of recombinant adeno-associated virus. …