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

Tet-on systems for doxycycline-inducible gene expression

AT Das, L Tenenbaum, B Berkhout - Current gene therapy, 2016 - ingentaconnect.com
… A next step in adenoassociated virus-mediated gene therapy for … tetracycline-dependent
transactivator correlates with long-term doxycycline-regulated transgene expression

A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses

A Chtarto, M Humbert-Claude, O Bockstael… - … therapy Methods & …, 2016 - cell.com
… -sensitive doxycycline-inducible adeno-associated virus (AAV) … regulatable system is the
tetracycline-dependent (tet) system. … Since long-term exposure to antibiotics is undesirable, …

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-…

Immune-evasive gene switch enables regulated delivery of chondroitinase after spinal cord injury

ER Burnside, F De Winter, A Didangelos, ND James… - Brain, 2018 - academic.oup.com
… switch, utilizing a chimeric transactivator designed to evade T cell … long term administration
would have any additional advantage over acute transient delivery. Doxycycline-regulated Tet…

A drug-tunable gene therapy for broad-spectrum protection against retinal degeneration

CP Santiago, CJ Keuthan, SL Boye, SE Boye… - Molecular Therapy, 2018 - cell.com
… of diseases characterized by the irreversible loss of light-sensitive … , we generated an
adeno-associated virus vector named … that long-term treatment of RPF2 did not alter retinal …

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 …

[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