Sentences with phrase «of gene therapy»

So far, scientists have carried out more than 350 clinical trials of gene therapy, and not one has been an unqualified success.

The new system is similar to those used for other forms of gene therapy.

This study, which is ongoing, assesses the feasibility and efficacy of gene therapy in this indication.

The success is a much - needed shot in the arm to the field of gene therapy, which has recently struggled with safety concerns.

The new method may make some types of gene therapy easier and could be a boon for researchers hoping to control gene activity in animals, scientists say.

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

Recently, studies have been done to examine the use of gene therapy to treat type I diabetes in dogs.

So a new wave of gene therapy research is turning to nanoparticles to get the job done.

«This is phenomenal, because the promise of gene therapy is clear,» he says.

Unfortunately these requirements are not applicable for this sort of a gene therapy drug.

The topic of gene therapy is presented with equal magnitude in the game.

The treatment is delivered just once, a facet of gene therapy that poses unique pricing questions in an industry fueled by steady payments for chronic therapies.

Plus, its treasure trove of DNA sample data from millions of customers is just the kind of testing data many of the next generation of gene therapies need.

The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.

A major goal for gene sequencing has always been as a platform for the design and implementation of gene therapy.

Because of the urgent needs of a large number of cancer patients, most of these gene therapy trials are directed against cancer.

Provided that the risk - to - benefit ratio of gene therapy can be improved significantly, a potential cure could have a dramatic impact on the sensibilities of a large slice of humankind.

The researchers conclude this technique could eventually lead to new ways to prepare vast numbers of cells for the coordinated manufacture of gene therapies.

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

The authors looked to the future of gene therapies, and the challenges of delivering these complex treatments to patients.

The goal of gene therapy is to replace damaged genes with ones that work to address a root cause of cancer: damage to DNA.

Because of the inherent nature of gene therapy, a single dose, phase 1 trial has the potential to save lives.

And because each cell has its own unique set of mutations, we can not solve them with the methods of gene therapy in the adult organism either today or tomorrow.

Right now, it is kind of aiming for the low hanging fruit of gene therapy.

The announcement last month that a fifth child who received gene therapy for an immune system disease has developed leukemia was the latest blow to the field of gene therapy.

Women were not included in the first trial of gene therapy, which was mainly concerned with proving the safety of the technique.

The concept of gene therapy is far from new, with initial studies performed over 20 years ago (1).

In the US, scientists are already experimenting with other forms of gene therapy for cancer.

Lysogene is a pioneer in the basic research and clinical development of gene therapy for neurodegenerative disorders.

That is why in May 2009 the American Society of Gene Therapy officially changed its name to the American Society of Gene & Cell Therapy.

The clinical trial of nine adult hemophilia B patients, aged 18 to 52 years, used a single dose of a gene therapy product engineered to enter patients» liver cells and direct the production of the blood clotting factor that they lack.

The RAC model of diverse committee membership and open meetings has ensured a public voice in the review of the safety and ethics of gene therapy research among academic and industrial investigators.

-- The NEJM published updates from two early - stage Bluebird Bio (NASDAQ: BLUE) studies of a gene therapy for the rare disease beta - thalassemia, which requires patients to... Next Page»

New insights into specific gene mutations that arise in this often deadly form of brain cancer have pointed to the potential of gene therapy, but it's very difficult to effectively deliver toxic or missing genes to cancer cells in the brain.

Concerns have been raised about the safety of gene therapy in the past, not least about links between the viruses used to transfer the genes and disease.

The company also plans to explore potential applications of its gene therapy platform in other indications in orphan ophthalmology.

Genetics writer Ricki Lewis uses Haas» story to bookend the checkered history of gene therapy — the idea of replacing a defective gene with a working one in order to knock out disease.

Ribozymes developed in our research program have been developed to target HIV and these have been inserted into human blood progenitor cells via a retroviral vector as part of a gene therapy program for the treatment of HIV infection.

The study's variable results to date raise questions about which factor is most important to the success of gene therapy for any given individual with WAS: the number of WAS gene copies the vector inserts into the patient's cells, the number of modified cells given to a patient, or how effectively the patient's native blood system is eliminated before the modified cells are infused.

As director of the Gene Therapy Program, she is working to extend gene therapy treatments to new diseases and is actively involved in clinical trials for genetic diseases.

The trial, sponsored by Généthon, will include 17 patients in four centres in Europe, and will assess safety and therapeutic efficacy of the gene therapy product developed by Généthon.

Intellia Therapeutics has a significant pipeline of gene therapies that explore knocking out «bad genes» and often replacing them with «good genes.»