Sentences with phrase «gene therapy also»

But gene therapy also needs to be carefully done and, ideally, two independent gene - manipulation systems would be used to ensure that stem cells remain firmly in control of clinicians.

Gene therapy also has the potential to cure other forms of vision impairment that are not inherited genetic mutations.

What's more, Discher says, unpublished work from his lab suggests that adding the molecular passports to viruses that deliver genes in gene therapy also helps them avoid immune detection.

Not exact matches

And Pfizer also struck a broad research pact with Spark Therapeutics ($ ONCE) covering gene therapy late last year.

Pfizer says it will also make some new hires for immuno - oncology and gene therapy, two fields where it recently completed major deals.

The company is also seeking deeper inroads into rare diseases, with a number of assets in late - stage development, and last year it scored a major regulatory win when Europe approved its «bubble boy syndrome» gene therapy Strimvelis.

Companies are also finding that using M&A to acquire R&D capabilities is an effective way to build positions in emerging technologies such as gene therapy and biosimilars.

The scientists are also experimenting with gene therapy, using a harmless virus to deliver a normal copy of the normal CIB2 gene to baby mice that have the mutated version.

Gene therapy and stem cells are also being explored as ways to restore sight, but a drug would be simpler and any side effects should be reversible, says Kramer.

These human genes were also protective against alpha - synuclein - induced death, suggesting that they could be worth testing as gene therapy treatments for Parkinson's disease, Lu says.

The laboratories also collaborated to successfully prove that reversing a protein deficiency through gene therapy is effective in improving and stabilizing SMA in a large animal model.

Duan also provided significant expertise from a long history of work on gene therapy for neuromuscular disorders.

The technique has also raised hope for more powerful gene therapies that can delete or repair flawed genes, not just add new genes.

It may also advance gene therapy for this disorder as well.»

These deletions also may extend to neighboring genes, an event known as «collateral lethality,» which may create new options for development of therapies for several cancers.

Since patients (and mice) with Usher 1c also have balance problems caused by hair - cell damage in the vestibular organs, the researchers also tested whether gene therapy restored balance.

«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.»

The work also shows we can alter development by changing the way genes are programmed, sidestepping the need for gene therapy.

An mRNA drug would also be easier to control than traditional gene therapy.

This achievement will also contribute to accelerating the research applications of RNAi such as to the development of RNA - based next - generation drugs, for example as gene therapy to suppress the production of a disease - causing protein.

However, Holt's study also showed that gene therapy with TMC2 could compensate for loss of a functional TMC1 gene, restoring hearing in the recessive deafness model and partial hearing in the dominant deafness model.

In the recessive deafness model, gene therapy with TMC1 restored the ability of sensory hair cells to respond to sound — producing a measurable electrical current — and also restored activity in the auditory portion of the brainstem.

It could also be relevant to clinical applications — it treats sequence uniqueness as a high priority and thus minimises the risk of potentially unwanted gene modifications, which must be avoided at all costs in gene therapy,» says Graf.

Such a treatment might be combined with a gene therapy approach, also being pursued by her team and other groups, delivered directly to the brain to curtail mHTT expression.

Unlocking the secret to hypertrichosis could lead to gene therapies not just for people with too much hair but also for some of those with too little.

The link to autism suggests that a therapy that resets gene behavior in fragile X syndrome may also ease other developmental disorders.

However, the researchers believe that these results are proof of concept that gene therapy is a valid strategy against aplastic anemia; this therapy could also be applied to other genes — besides from telomerase — if a causal role for those other forms of the disease was discovered.

The nanotechnology approach that is being tested for inhibitor control could also improve the haemophilia treatment that is now at the cutting edge of clinical research: gene therapy.

«The good news is that this finding predicts that patients missing either gene should be sensitive to new therapies targeting focal adhesion enzymes, which are currently being tested in early - stage clinical trials,» says Shaw, who is also a member of the Moores Cancer Center and an adjunct professor at the University of California, San Diego.

But women are also likely to be the first recipients of gene therapies to improve fertility.

«In the group that was not treated with the gene therapy, the majority of the animals die from aplastic anemia, and they also die much sooner.»

As in the former case, after being treated with telomerase gene therapy «the telomeres in the peripheral blood in these mice also lengthened and the number of blood cells increased considerably,» write the authors.

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.

Dr. Oesterreich and her colleagues propose that future studies look at offering a combined therapy that, along with aromatase inhibitors, also introduces drugs that modify the epigenome to prevent or delay the cancer from repressing cancer - killing genes.

Using cell cultures, the research team also found two ways to prevent the toxicity caused by alpha - synuclein: gene therapy that forced the neurons to make more TOM20 protein protected them from the alpha - synuclein; and a protein that was able to prevent alpha - synuclein from sticking to TOM20 prevented alpha - synuclein's harmful effects on mitochondria.

The first people to be treated with a gene therapy had ADA - SCID, also called «bubble boy disease», and some later got leukaemia, probably because the virus carrying the new genes also switched on cancer genes.

Experimental approaches such as gene therapy are also being investigated, but Dr. Rudnicki's research suggests that these approaches will have to be modified so that they target muscle stem cells as well as muscle fibres.

By targeting the fundamental DNA of the gene, the zinc finger therapy also has the advantage over other potential Huntington's therapies of needing less frequent treatments.

Rivals such as Pfizer and Sanofi are also investing, and overall financing for gene and gene - modified cell therapies reached $ 1 billion in the first quarter of 2017, according to the Alliance of Regenerative Medicine.

If you could figure out which modifications work, and if you could also find some gene - therapy technique for delivering those changes to the host cells, you could in principle make a person (or livestock, or any other creature) inherently resistant not to just one virus but to all viruses, even those that have not been discovered.

Shire CEO Flemming Ornskov - who has a large conventional haemophilia business and is also chasing Biomarin and Spark in hunting a cure for the bleeding disorder - sees both the opportunities and the difficulties of gene therapy.

But gene therapy companies - which also include Bluebird Bio, BioMarin, Sangamo and GenSight - may need new business models.

The gene therapy was also designed to inhibit action of the myostatin gene, which stops muscle cells from growing.

The researchers also hope that by advancing the understanding of which genes play a role in DLB, their results will aid in the development of targeted therapies.

The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease.

Patients who were using full - time ventilator support at the start of the study also showed some benefit from gene therapy.

An 18 - year - old also died following a reaction to a virus used in gene therapy for a liver condition.

The team also set out to deplete the large supply of MDSCs, and to explore whether that would allow immune - mediated gene therapy to eradicate glioblastoma in mouse models.

Along with differences in gene alterations, bladder cancer patients also show differences in how well they respond to certain therapies.

She also writes about research on gene therapy and cancer.