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
Widespread use of gene therapy technologies is limited in part by the lack of small genetic
switches with wide dynamic ranges that control transgene expression without the …

Immune responses to adeno-associated virus vectors

AK Zaiss, DA Muruve - Current gene therapy, 2005 - ingentaconnect.com
One of the biggest challenges in optimizing viral vectors for gene therapy relates to the
immune response of the host. Adeno-associated virus (AAV) vectors are associated with low …

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

D Stone, M Aubert, KR Jerome - Gene Therapy, 2023 - nature.com
Over 15 years after hepatotoxicity was first observed following administration of an adeno-
associated virus (AAV) vector during a hemophilia B clinical trial, recent reports of treatment …

Complement is an essential component of the immune response to adeno-associated virus vectors

AK Zaiss, MJ Cotter, LR White, SA Clark… - Journal of …, 2008 - Am Soc Microbiol
Adeno-associated virus (AAV) vectors are associated with relatively mild host immune
responses in vivo. Although AAV induces very weak innate immune responses, neutralizing …

Conditional control of mammalian gene expression by tetracycline-dependent hammerhead ribozymes

K Beilstein, A Wittmann, M Grez, B Suess - ACS synthetic biology, 2015 - ACS Publications
Robust synthetic devices are requisite for the construction of synthetic genetic circuits and
important scientific and technological tools to control cellular processes. We developed …

Immune responses to adenovirus and adeno-associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the …

PR Lowenstein, RJ Mandel, W Xiong… - Current gene …, 2007 - ingentaconnect.com
Researchers have conducted numerous pre-clinical and clinical gene transfer studies using
recombinant viral vectors derived from a wide range of pathogenic viruses such as …

In vivo gene regulation using tetracycline-regulatable systems

K Stieger, B Belbellaa, C Le Guiner, P Moullier… - Advanced drug delivery …, 2009 - Elsevier
Numerous preclinical studies have demonstrated the efficacy of viral gene delivery vectors,
and recent clinical trials have shown promising results. However, the tight control of …

Synthetic transcription factor engineering for cell and gene therapy

B Bhatt, P García-Díaz, GW Foight - Trends in Biotechnology, 2023 - cell.com
Synthetic transcription factors (synTFs) that control beneficial transgene expression are an
important method to increase the safety and efficacy of cell and gene therapy. Reliance on …

Lentiviral vectors for use in the central nervous system

J Jakobsson, C Lundberg - Molecular Therapy, 2006 - cell.com
Lentiviral vectors have been used extensively as gene transfer tools for the central nervous
system throughout the past decade since they transduce most cell types in the brain …

Biosafety of recombinant adeno-associated virus vectors

DJ Dismuke, L Tenenbaum… - Current gene …, 2013 - ingentaconnect.com
It is hoped that the use of gene transfer technology to treat both monogenetic and acquired
diseases may soon become a common therapy option in medicine. For gene therapy to …