Not exact matches
Anderson and others are already exploring new
gene -
delivery methods, including nanoparticles, to
better deliver
gene - editing therapies to target tissues.
This makes possible a wide range of biomedical applications such as tissue engineering as
well as drug and
gene delivery.
«
Gene therapy restores hearing in deaf mice, down to a whisper: Improved
delivery vector
better penetrates the inner ear, also restores balance in a mouse model of Usher syndrome.»
Initial tests on mice showed the hybrid virus was very efficient: the
gene it carried was active in 24 per cent of airway cells after two months, a far
better proportion than achieved by other
delivery methods (New Scientist, 10 March 2001, p 19).
With the
best - performing particles, the researchers reduced
gene expression by more than 50 percent, for a dose of only 0.20 milligrams per kilogram of solution — about one - hundredth of the amount required with existing endothelial RNAi
delivery vehicles.
In large animal models of heart failure, the researchers found that
gene therapy
delivery of high dose SUMO - 1 alone, as
well as SUMO - 1 and SERCA2 together, result in stronger heart contractions,
better blood flow, and reduced heart volumes, compared to just SERCA2
gene therapy alone.
We have demonstrated that our liposomes can mediate in vivo target mRNA
delivery as
well as
gene disruption using an entirely RNA - based approach.
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.
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»).
«Our idea is to hone in as much as possible on targets and refine therapies and make everything as focal as possible, both in terms of the
genes and proteins as
well as the spatial
delivery,» she said.