Sentences with phrase «gene therapy technologies»

Her postdoctoral research was conducted with a team of CNRS researchers in Paris, France, where she employed biochemical and gene therapy technologies to investigate the impact of protein aggregation in Parkinson's disease.

Kotterman was 4DMT's first hire after working on its gene therapy technology as a graduate student in Schaffer's lab.

Tissue engineering combined with gene therapy technology has the potential to manage the repair of defective articular cartilage.

Through the collaboration, Lysogene, UMMS and AU will develop IND - supporting preclinical studies in GM1 - gangliosidosis using AAV gene therapy technology.

In collaboration with the University of Massachusetts Medical School (UMMS) and Auburn University (AU), Lysogene is developing IND - supporting preclinical studies using AAV gene therapy technology to translate the initial proof of concept into a viable drug development program to the benefit of patients in urgent need.

Not exact matches

Editas, a startup based in Cambridge and founded in November 2013, wants to eventually use the technology to treat disease by coming up with therapies that can modify faulty disease - causing genes.

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.

On the supply side, evolving technology and industry dynamics have driven cutting edge research and investments in diverse areas like gene therapy, early detection / diagnostics, personalized medicine and telemedicine, to better meet the demand for cost efficient and accessible healthcare.

The Chiesi fund will consider a wide range of opportunities, from traditional small molecules and biologics, to gene therapies and cell therapies, to diagnostic and disease management technologies.

New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.

«The event, the fourth of its kind, seeks to raise global awareness and create a forum for collaboration around the wide array of powerful and promising cell therapies, gene therapies, and immunotherapies emerging from medical institutions around the world, as well as the impact new technology will have on humanity and society,» a press release by the Cure Foundation explains (h / t Christian Post).

«Steep funding cuts for the federal health agencies are counterproductive at a time when innovative research is moving us closer to identifying solutions for rare diseases, new prevention strategies to protect Americans from deadly and costly conditions, advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.»

In what could be the start of a new era of do - it - yourself gene therapy, the CEO of a biotech startup last month skipped town to undergo a very public round of anti-aging therapy in Latin America, reports MIT Technology Review.

For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease: sickle cell disease.

Being able to acquire new technologies, as well as becoming more innovative internally by venturing into new research areas, such as stem cell and gene therapy research, have allowed Genzyme to maintain its edge.

However, in the wake of fatalities from gene therapy and other technologies, as well as the potential for cancers associated with stem cell transplants, governments are understandably nervous about safety issues — not to mention the ethical maze of tinkering with fledgling life.

The roadmap outlines future research directions toward the goal of enhancing human radioresistance, including upregulation of endogenous repair and radioprotective mechanisms, possible leeways into gene therapy in order to enhance radioresistance via the translation of exogenous and engineered DNA repair and radioprotective mechanisms, the substitution of organic molecules with fortified isoforms, the coordination of regenerative and ablative technologies, and methods of slowing metabolic activity while preserving cognitive function.

«It's as close to a definitive clinical success with gene therapy as one could have at this point in the development of the technology,» says genetic engineer Richard Mulligan of Harvard University.

«The technology will get better,» agrees cancer gene therapy researcher Carl June of the University of Pennsylvania.

Fisher and his colleagues are pioneering this approach and have already reported success in preclinical experiments utilizing UTMD technology and mda - 7 / IL - 24 gene therapy in prostate and colorectal cancer models.

Their research has created the technology known as gene targeting, now used in virtually all areas of biomedicine — from basic research to the development of new therapies.

Furthermore, the same technologies used to create genetic models of cancer show promise as gene therapies capable of repairing mutations that lead to a range of diseases.

Intellia has exclusive access to Caribou's CRISPR - Cas9 technology for the development of new human gene and cell therapies as well as anti-viral therapies.

Intellia is developing human gene and cell therapies for both ex vivo and in vivo applications using CRISPR - Cas9 gene editing technology.

PNP Therapeutics, Inc. — a company based on a gene - therapy technology developed by scientists at Southern Research Institute and the University of Alabama at Birmingham (UAB)-- has found an approach that has abolished otherwise unmanageable human cancers in preclinical rodent studies.

The technology also improves cell culture systems used to produce therapeutic proteins, vaccines, and gene therapy vectors.

Using advanced high - throughput technology, the laboratory has developed gene signature profiles to help predict disease recurrence and response to medical therapies.

Caribou recently cofounded Intellia Therapeutics for the development of human gene and cell therapies based on their proprietary CRISPR - Cas9 technology platform.

Poseida Therapeutics is translating best - in - class gene engineering technologies into lifesaving cell therapies.

The goal of the Program in Human Gene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseaGene Therapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diTherapy is to develop gene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene transfer technologies and use them for hepatic gene therapy for the treatment of genetic and acquired diseagene therapy for the treatment of genetic and acquired ditherapy for the treatment of genetic and acquired diseases.

The CRISPR - Cas technology can be used to screen for druggable targets and to develop gene and cell therapies for unmet medical needs.

Intellia will utilize Caribou's proprietary CRISPR - Cas9 gene editing and repair technology platform in the development of new therapies targeting a variety of genetic - based diseases.

One form of gene therapy involves the direct repair of a defective gene, using genome - editing technology such as CRISPR - Cas9.

He provides advice and recommendations on issues relating to genetic testing, screening, gene patents, genetic privacy, stem cell therapy and the translation of new knowledge and technologies to improve in health services.

To enable scaling long term, we have entered into an agreement with Cell Therapy Catapult Services, a research organization specializing in the development of technologies which speed the growth of the cell and gene therapy inTherapy Catapult Services, a research organization specializing in the development of technologies which speed the growth of the cell and gene therapy intherapy industry.

It was created to support development and commercialization of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy.

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).

With her recent groundbreaking findings in the field of RNA - mediated regulation based on the CRISPR - Cas9 system, E. Charpentier has laid the foundation for the development of a novel, highly versatile and specific genome editing technology that is revolutionizing life sciences research and could open up whole new opportunities in biomedical gene therapies.

His track record includes biologics and gene therapies, which were new technologies to both U.S. and European regulators at the time of their development.

Among candidate technology areas for the prospective Catapult centres was a young but promising field of cell and gene therapy.

Gene and cell therapies have made important medical advances over the past three decade, developing technologies and testing novel therapies in multiple human clinical trials of many diseases.

This gene editing technology continues to become more sophisticated, and several HD research teams are adapting it to the challenges of HD therapy.

Her game - changing technology takes a mysterious bacterial genetic code and transforms it into a powerful tool for cutting and pasting bits of genetic material — meaning not only could the entire field of gene therapy be revived, but her genome - editing tool could one day be used to treat a range of diseases, from cancer and AIDS to hereditary disorders like Down syndrome and Huntington disease.

xviii This technology is still in the early stages of development and continued research is vital in order to translate the technology into the clinic for PID gene therapy as soon as possible.

CCC's journals cover a wide range of current research serving the fields of oncology research, gene expression & therapy, pain research, cell transplantation, alternative medicine, and technology.

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.

Zinc finger technology is one of the hottest areas now in everything from gene therapy for HIV to increasing crop yield, to therapeutic protein production, to the great hope for stem cell therapy.

This is an issue that can be dealt with and we have the technology to do that in the form of gene therapy,» says senior author Dr. Ronald G. Crystal, chief of the Division of Pulmonary and Critical Care Medicine at NewYork - Presbyterian Hospital / Weill Cornell Medical Center, and the Bruce Webster Professor of Internal Medicine and Professor of Genetic Medicine at Weill Cornell Medical College.

Performs genome - wide profiling of vector integration sites as readout of cell growth at clonal level, in basic research studies and technology development, in preclinical safety studies and in gene therapy treated patients.

The Company's transformative gene repair technology presents a promising opportunity to create novel therapies that address the significant unmet need in the treatment of serious genetic neuromuscular diseases.