Sentences with phrase «gene delivery in»

Cell - Specific Targeting Strategies for Electroporation - Mediated Gene Delivery in Cells and Animals.

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].

To correct RPE65 mutation damage, scientists are evaluating drugs, cell transplants and gene delivery in Rpe65 knockout, transgenic and spontaneous mutant mice, and spontaneous Rpe65 - mutant dogs.

Not exact matches

Gene therapy involves the delivery, using a modified virus, of a healthy copy of a gene to make up for one that's deficient in a way that causes diseGene therapy involves the delivery, using a modified virus, of a healthy copy of a gene to make up for one that's deficient in a way that causes disegene to make up for one that's deficient in a way that causes disease.

Saam's delivery is relaxed, almost singsong, which is in direct contrast to his TV - radio partner, GENE KELLY (39, partisan), who tends to be loud and shrill.

HMOs vary between pre-term and full term birth, vaginal deliveries and planned Cesarean births (reviewed in [20]-RRB-, and even between mothers with different types of «secretor» genes [21].

«Gene - delivery system prevents vision loss from inherited eye disease: Initial testing in animal models holds promise for treating Leber congenital amauroisis and other congenital visual disorders.»

«New gene - delivery therapy restores partial hearing, balance in deaf mice.»

The team now plans to improve their gene - delivery technique in an attempt to reach an even greater proportion of hair cells.

To use viruses as delivery vehicles for gene therapy, researchers take all the harmful and replicative genes out of the virus and put in the therapeutic genes they want to deliver.

«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.»

When oxygen is scarce, however, HIF - 1α starts to accumulate and turns on the expression of a host of genes that assist in promoting and increasing oxygen delivery to hypoxic regions.

Muscle biologists Qi Long Lu and Terence Partridge at the Medical Research Council Clinical Sciences Centre in London, U.K., and their colleagues decided to combined the antisense strategy with a chemical often used in gene therapy because it is known to improve delivery of DNA into cells.

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).

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.

«To date, this type of system has only been used in humans with viral methods of gene delivery, of which the safety profiles are still heavily in debate,» says Betty Tyler, associate professor of neurosurgery at Johns Hopkins.

This has limited the application of gene therapy, along with other concerns in the field, like the safety of the delivery vector.

Using molecular scissors wrapped in a greasy delivery package, researchers have disrupted a gene variant that leads to deafness in mice.

To confirm the links, the group harnessed cutting edge techniques in genetic engineering and viral gene delivery to switch on neurons in the front of healthy mouse brains.

And many companies began to crumble: While gene silencing was simple in worms, mammals proved to be far more complex — and safe drug delivery emerged as a major problem.

«We are hopeful that a systemic dose and delivery of gene therapy would broadly affect whole - body strength and function, and we are working on a host of projects for such systemic delivery in Pompe and other neuromuscular conditions,» said Barry Byrne, M.D., Ph.D., director of the Powell Gene Therapy Center, a professor of pediatrics in the UF College of Medicine, and a member of the UF Genetics Institgene therapy would broadly affect whole - body strength and function, and we are working on a host of projects for such systemic delivery in Pompe and other neuromuscular conditions,» said Barry Byrne, M.D., Ph.D., director of the Powell Gene Therapy Center, a professor of pediatrics in the UF College of Medicine, and a member of the UF Genetics InstitGene Therapy Center, a professor of pediatrics in the UF College of Medicine, and a member of the UF Genetics Institute.

This block is caused by mutations of the gene encoding the γc cytokine receptor subunit of interleukin - 2, -4, -7, -9, and -15 receptors, which participates in the delivery of growth, survival, and differentiation signals to early lymphoid progenitors.

There is a size limit in terms of what you can package into the gene delivery vehicle, and otoferlin is too large.

In addition to being the vector of choice for gene delivery to a variety of cell types, now you know that AAVs can provide a powerful boost to genome editing experiments.

Scientists know which part to replace, but the real problem, again, is delivery: «We would have to wrap the gene in such a way that it could be picked up by muscle cells and incorporated into them,» Davies said.

Asuri, Prashanth, et al. «Directed evolution of adeno - associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells.»

In particular, the advanced drug delivery systems he has developed provide new methods for nanoparticulate drug delivery, non-viral gene therapy, siRNA delivery, and vaccines.

Enhancing muscle membrane repair by gene delivery of MG53 ameliorates muscular dystrophy and heart failure in d - Sarcoglycan - deficient hamsters.

Lombardo A, Genovese P, Beausejour CM, Colleoni S, Lee YL, Kim KA, Ando D, Urnov FD, Galli C, Gregory PD, Holmes MC, Naldini L. Gene editing in human stem cells using zinc finger nucleases and integrase - defective lentiviral vector delivery.

It is hoped that the delivery of the missing SGSH gene provides a permanent source of functional enzyme in the brain that reverses phenotypic abnormalities of CNS neural cells.

Request a custom bibliography search or select from our list of citations available on - line: Select one Agents Administered Biotechnology / Protein & Peptide Delivery Cancer Research Cardiovascular Research Endocrinology Gene Therapy Immunology Neuroscience Pharmacology Physiology Route of Agent Administration Toxicology / Teratology In Vitro Use Nude Mice Neonates

I don't believe that the problem of in vivo gene delivery by vector has been solved.

«The failure rate to deliver drugs to CNS is unfortunately very high, so any new methods of drug, protein and gene delivery should be welcome,» says Inder Verma, Ph.D., a professor in the Laboratory of Genetics and senior author of the study published in the Proceedings of the National Academy of Sciences.

Investigating polymer thiolation in gene delivery.

New delivery technique used to create a glowing adult mouse potentially opens the door for in vivo gene therapy

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 alonIn 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 alonin stronger heart contractions, better blood flow, and reduced heart volumes, compared to just SERCA2 gene therapy alone.

To use viruses as delivery vehicles for the gene therapy, the researchers took all the harmful genes out of the virus and put in the beneficial genes in.

These features enable gesicles to knock out genes with high efficiency and in a broader range of cell types than plasmid - based delivery methods.

«This is important biotechnologically, because if you look at if from the angle of genome editing, the delivery of small genes into cells is much easier than the delivery of large genes,» said Rotem Sorek of the Weizmann Institute of Science in Israel who was not involved in the work.

Basically, the cells to be reprogrammed are removed from their normal culture system and exposed to the genes (in a gene delivery system), then put back into a new culture system.

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

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).

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.

This molecule brings a revolutionary technology platform for genetic engineering in vertebrates, including gene discovery in model species and for therapeutic transgene delivery for possible human applications.

We have demonstrated that our liposomes can mediate in vivo target mRNA delivery as well as gene disruption using an entirely RNA - based approach.

By eliminating HIV's ability to replicate (a feat accomplished in the mid 90s), Dr. Verma's group created a vector, or carrier, that is now used worldwide for gene delivery.

LA JOLLA, CA — Using a gene therapy delivery system developed in the laboratory of Inder Verma at the Salk Institute for Biological Studies, an international team of researchers successfully treated two...

Sustained high - level expression of human factor IX (hFIX) after liver - targeted delivery of recombinant adeno - associated virus encoding the hFIX gene in rhesus macaques.

Researchers describe how a bacterial infection - based protein delivery strategy can mediate effective and safe gene editing in human pluripotent stem cells.

Delivery: getting the CRISPR - Cas9 machinery to enter many neurons in the brain and remove the HD gene from each.