Sentences with phrase «rpe65 gene vector»

I won't reveal yet who my favorites are, but I will say that these young scientist - founders came up with very creative solutions for preventing infections in some common surgeries, tackling resistance in targeted antibody drugs, improving gene vectors for cell therapies, helping the vision - impaired «see» faces and better read their environments, imaging hard - to - see spots in the lungs and other organs, improving genetic risk analysis, and expediting the logistical operations of hospitals.

Next Wave asked: If research on gene vectors can be used not only to further gene therapy but also — potentially — to aid in the development of biological weapons, should scientists be concerned about the potential applications of their research in genetics (as Joseph Rotblat suggested in a 19 Nov 1999 editorial in Science magazine?)

Pang and his team hypothesized that the RPE65 gene vector they were using did not transfect the few cones that survive into later LCA stages.

This notion served later as a model for viral gene vectors in genetic engineering.

Not exact matches

«Recombinant DNA technology», «DNA cloning», «molecular cloning» or «gene cloning» all describe the process of transferring a DNA fragment from one organism to a self - replicating genetic element (a cloning vector) such as a bacterial plasmid, enabling the fragment to be propagated in an alien host.

«A German athletic coach was found attempting to obtain Repoxygen, a gene transfer vector that expresses the erythropoietin gene [and boosts red blood cell production].

This fact is rooted in a packaging problem: the gene encoding FIX is much smaller than the gene for FVIII, and better fits into a vector, usually a harmless virus, designed to deliver therapeutic genes to a patient.

Bacteriophages, or viruses that infect bacterial pathogens, may also act as vectors of virulence or of antibiotic resistance genes, ultimately making bacterial disease worse for the host.

We use viruses that have been gutted of all their viral genes and use them as vectors.

«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.»

While previous vectors have only been able to penetrate the cochlea's inner hair cells, the first Nature Biotechnology study showed that a new synthetic vector, Anc80, safely transferred genes to the hard - to - reach outer hair cells when introduced into the cochlea (see images).

The vector then unloads its genetic material containing the therapeutic human gene into the target cell.

A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells.

AAV1 is considered safe as a viral vector and is already in use in human gene therapy trials for blindness, heart disease, muscular dystrophy and other conditions.

The aim is to introduce into the shuttle a viral vector loaded with the gene that the cells of these patients lack, with a particular focus on targeting neurons.

Someone would have to provide expertise in handling viruses — specifically, a virus to serve as a vector, or Trojan horse, to cart algal genes into mammalian cells.

After that was settled, gene therapists still had to find a suitable virus, or vector, to carry replacement genes into human cells without inciting a damaging or deadly immune response.

A company called Clontech — also based in Palo Alto — makes virally derived vectors that carry the gene for GFP into cells.

The vectors contain the gene for GFP spliced to the gene for the protein that is being tracked.

Gene therapy researchers have found that a popular viral vector used to insert disease - curing genes into mice has a dangerous down side: it can apparently trigger liver tumors.

In four tumors, the vector mapped to a section of chromosome 12 and turned on several genes implicated in cancer, the team reports tomorrow in Science.

A virus that has shown promise as a vector for human gene therapy causes liver tumors in neonatal mice.

This group is well known for its design of retroviral vectors for efficient gene transfer and it seemed like a good idea for me to go there for a year and exchange knowledge and expertise.

Wilson famously led a gene therapy study with an adenovirus vector that killed a patient, Jesse Gelsinger, in 1999 — a major setback for the entire field.

With this in mind, independent teams led by Scott and Herzog took the conventional viral - vector approach to inducing tolerance through gene therapy.

They directly injected into the cerebrospinal fluid of a mouse model of Alzheimer's — adult animals in which plaques were well established — viral vectors carrying genes for one of the three APOE variants or a control protein.

«The vectors developed and characterized in this study demonstrate unique and potent biology that justify their consideration for gene therapy applications,» Vandenberghe says.

Thus, gene drive could be used to reduce malaria transmission in humans — or in endangered birds (see image, above)-- by making the mosquito vectors incapable of spreading the malaria parasite or even eliminating the insects altogether.

Single - cell mouse embryos were infected in vitro with recombinant lentiviral vectors to generate transgenic mice carrying the green fluorescent protein (GFP) gene driven by a ubiquitously expressing promoter.

Still, the standard form of liver - targeted gene therapy carries a range of potential complications, including the risk of harmful mutations and of the body mounting an immune response against the viral vectors used to carry the correct forms of the defective genes responsible for haemophilia.

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

Because viral vectors have been stripped of their own genes and, thereby, of their ability to replicate, they are no longer infectious.

Such vectors have become critical tools for delivering genes to treat disease or to label neurons and their connective fibers with fluorescent colors to map out their locations.

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

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.

The new AAV vectors can also deliver genes that code for colorful fluorescent proteins; such proteins are useful in identifying and labeling cells.

The vectors are customizable and could potentially be used as part of a gene therapy to treat neurodegenerative disorders that affect the entire central nervous system, such as Huntington's disease, or to help map or modulate neuronal circuits and understand how they change during disease.

Since the discovery of the genetic basis for cystic fibrosis in 1989, scientists have developed a variety of viral and non-viral vector systems for delivering a corrected CFTR gene back into lung cells.

Using support vector machine recursive feature elimination, the researchers identified three gene expression signatures that predicted therapy responses.

«This methodology is very important because it allows you to very quickly integrate almost any piece of DNA in these vectors,» remarks Bellen, who says this technology also allows scientists to put single point mutations in a gene and then reinsert the gene into a genome as well as tagging a segment of DNA with fluorescent markers.

Specifically, the new results boost knowledge about the effects of the viral vector used, adeno - associated virus, which has been successfully used in gene therapy for the eye since 2008.

«For our new systemic viral vectors — AAV PHP.S and AAV PHP.eB — there are many potential uses, from mapping circuits in the periphery and fast screening of gene regulatory elements to genome editing with powerful tools such as CRISPR - Cas9,» says Gradinaru.

(Adeno - associated virus or AAV has been approved in Europe as a vector for gene therapy, and is being explored in the United States for clinical trials.)

Last year, the Li lab published a paper in Journal of Clinical Investigation showing that CRISPR - Cas9 gene editing, delivered by viral vector, can reverse signs of HD in a mouse model.

So in an attempt to cure mice of hepatitis B, Kay's team flooded their livers with double - stranded RNA from a gene transfer vector.

1999 18 - year - old Jesse Gelsinger dies following an immune reaction to the virus vector used to insert the corrected gene.

In the study, which began in 2011, an adeno - associated virus vector was used to carry a functional copy of the affected gene to muscle cells in the diaphragms of nine patients.

The non-pathogenic vectors serve as gene shuttles to transport components of SIV, such as envelope proteins, into the target cells.

The results drew attention in part because they come from the lab of James Wilson at the University of Pennsylvania, who led a 1999 trial in which a teenager died from an immune reaction to a different gene therapy vector.

One subject self - infused two days after receiving the gene therapy vector.