On their quest to restore hearing through gene therapy, scientists have long sought ways to improve
gene delivery into hair cells.
Serap Günes (TUD)-- «Modification of vesicular stomatitis virus G protein for
targeted gene delivery into PSCA - positive tumor cells» (2007)
NGF is in fact viewed as a viable target for AD clinical trials with one group investigating NGF ex vivo
gene delivery in a Phase 1 trial with human patients aimed at stimulating cholinergic function and improving memory [123].
Asuri, Prashanth, et al. «Directed evolution of adeno - associated virus for
enhanced gene delivery and gene targeting in human pluripotent stem cells.»
The variety of new tools available for genetic manipulation now include lentiviral -
based gene delivery, and gene editing using CRISPR / Cas9, zinc finger nucleases (ZFNs) or transcription activator - like effector nucleases (TALENs).
Now, to enable
widespread gene delivery throughout the central and peripheral nervous systems, Caltech researchers have developed two new variants of a vector based on an adeno - associated virus (AAV): one that can efficiently ferry genetic cargo past the blood - brain barrier; and another that is efficiently picked up by peripheral neurons residing outside the brain and spinal cord, such as those that sense pain and regulate heart rate, respiration, and digestion.
Nanoparticles conjugated with two active ligands, one for its effective uptake and the other for it's binding or use to
gene delivery agents to deliver drug inside cells.
These alternate strategies include using small - molecule drugs that affect RNA metabolism or protein stability, as well as administering modified viruses for
therapeutic gene delivery (see «Getting a fix on SMA»).
The title of the paper is «The
Ideal Gene Delivery Vector: Chromallocytes, Cell Repair Nanorobots for Chromosome Replacement Therapy» and it is currently in press at the peer - reviewed Journal of Evolution and Technology (and is soon to be available online).
In
vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.
The lab is developing a
novel gene delivery system to produce anti-tumor and immunomodulatory antibodies in vivo for cancer treatment.
Important reports from the Weiner lab include the first DNA vaccine studied for HIV as well as for cancer immune therapy of cutaneous T cell lymphoma, the early development of DNA encoded genetic adjuvants, including IL - 12, advances in gene optimization, and advances in electroporation technologies resulting in
improved gene delivery.
A plasmid is like a
bacterial gene delivery truck; it is the primary way antibiotic resistance genes spread between bacteria.
The safety trial of the
new gene delivery method is now underway at Rush University Medical Center in Chicago.
The advent of molecular cloning, DNA sequencing and the many tools of molecular genetics and cell biology has given us sufficient knowledge of the basis for disease and the genes to target, but what has limited the application of gene therapy has been
efficient gene delivery systems.
Deal C, Balazs AB, Espinosa D, Zavala F, Baltimore D, Ketner G. Vectored
antibody gene delivery protects against Plasmodium falciparum sporozoite challenge in mice.
Scientists have uncovered variations in the surface structures of adenovirus species that may help us develop
better gene delivery tools
Perhaps, a molecule that could stimulate neurotrophic factors, such as NGF in vivo, would be more efficient, more safe and cost effective than
gene delivery directly into the brain.
Research Paper Fabrication of Positively Charged Fluorescent Polymer Nanoparticles for Cell Imaging and
Gene Delivery Lin Wei, Di Zhang, Xuanfang Zheng, Xuyao Zeng, Youlin Zeng, Xinbo Shi, Xin Su, Lehui Xiao Nanotheranostics 2018; 2 (2): 157 - 167.
The team is planning to continue with a range of investments in automation,
gene delivery technologies and work with Innovate UK to develop specialist treatment centres.
In addition, researchers examined the brain tissue of a study participant who had died and found robust growth of extensions from the dying cholinergic cells near the site of growth
factor gene delivery.
Dr. Rex
used gene delivery to provide long - term production of a modified form of erythropoietin (EPO), a hormone that induces red blood cell production but is also a neuroprotective cytokine.
Novartis will add an
AAV9 gene delivery platform and manufacturing facility through the $ 8.7 bn ($ 7bn) acquisition of AveXis.
In addition to revealing that biodegradable polymeric nanoparticles represent a promising mode
of gene delivery for glioma, the findings show that nonviral DNA delivery of HSVtk combined with administration of ganciclovir has potent antitumor effects.
«It is exciting to have found a way to selectively
target gene delivery to cancer cells,» says Quiñones - Hinojosa.
Robert A. Freitas Jr., «The
Ideal Gene Delivery Vector: Chromallocytes, Cell Repair Nanorobots for Chromosome Replacement Therapy,» J. Evol.
Meanwhile,
gene delivery systems other than retro - or lenti - viral ones, such as repeated transfection with plasmids and use of a non-integration virus, have been successfully applied in generation of iPS cells [8], [9].
Phrases with «gene delivery»