A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo
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 …
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 …
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
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 …
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 …
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 …
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 …
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 …
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 …
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 …
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 …
diseases may soon become a common therapy option in medicine. For gene therapy to …