Not exact matches
Now that the barrier
of editing
genes in the hospital setting has been broken, these companies and more have pipelines
of gene - editing innovation coming to a hospital near you in the next few years, as clinical
trials are approved and
new ways to cure formerly incurable diseases go mainstream in our lifetimes.
«Testing
of new rice varieties that have the SPIKE
gene is under way in multilocation
trials across several developing countries in Asia, including Indonesia.
They have discovered two
new genes implicated in two medical conditions and conducted one
of the cheapest clinical
trials in history, formulating a nutritional formula from pig brains that supplements a protein missing in a rare developmental disorder.
As CRISPR - Cas9 starts to move into clinical
trials, a
new study published in Nature Methods has found that the
gene - editing technology can introduce hundreds
of unintended mutations into the genome.
A one - time intravenous infusion
of the high dose
of gene therapy extended the survival
of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical
trial, according to a study published in the
New England Journal
of Medicine.
«Fibroblast growth factor receptor inhibitors are
new therapies being developed in clinical
trials for patients whose cancer cells have genetic alterations in this family
of genes,» says Roychowdhury, a member
of the OSUCCC — James Translational Therapeutics Program.
One clinical
trial involves the drug CGF166, a one - time
gene therapy, which, if proven successful in humans, could regenerate
new hair cells within the cochlea that can signal the part
of the brain that processes sound.
The
new method has been tested in greenhouse
trials to show that the more copies
of the
gene, the greater the resistance to soybean cyst nematode.
In 2011, Science's editors will be watching a smaller detector at the Large Hadron Collider called LHCb, which will study B mesons in great detail;
new techniques that should lead to the discovery
of many more
genes contributing to adaptation; an ignited fusion burn at the National Ignition Facility; broadly neutralizing antibodies, which are capable
of disabling a wide range
of viral variants; the first plug - in hybrid electric cars whose batteries are charged from a wall socket go on the market; and the results
of the first phase III
trial of a malaria vaccine.
Sangamo biochemist Philip Gregory notes that this Phase 1 clinical
trial is the first human test
of an entirely
new class
of drug that could turn any
gene on or off, depending on the disease.
«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.
A single injection
of a
new treatment has reduced the activity
of the
gene responsible for Huntington's disease for several months in a
trial in mice.
Less than a decade after a powerful
gene - silencing method — RNA interference, or just RNAi — was discovered, the field's pioneers have not only won the 2006 Nobel Prize in Physiology or Medicine but have also helped launch an entirely
new class
of drugs into human clinical
trials.
18 Drugs Aim to Silence Bad
Genes RNA interference helped launch an entirely
new class
of drugs into
trials...
As a
new generation
of gene therapy clinical
trials shows promise to cure or halt the progression
of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.
Beginning with the Nazi Doctors»
Trial at the 1946 Nuremberg
Trial (1), coverage includes publication
of Henry Beecher's «Ethics and Clinical Research» (2), The
New York Times exposure
of the public health service syphilis study in Macon County, Alabama (the infamous «Tuskeegee case»)(3), the University
of Pennsylvania / Gelsinger
gene transfer case, and The Washington Post series on international clinical drug testing abuses (4).
In human
trials, researchers remove some
of patients» T cells through a process similar to dialysis and then engineer them in a laboratory to add the
gene for the CAR so that the
new receptor is expressed in the T cells.
Gene therapy experts are heartened by the new results, which may counteract a wave of negative publicity after the death last year of a volunteer in a gene therapy trial at the University of Pennsylvania (ScienceNOW, 9 December 19
Gene therapy experts are heartened by the
new results, which may counteract a wave
of negative publicity after the death last year
of a volunteer in a
gene therapy trial at the University of Pennsylvania (ScienceNOW, 9 December 19
gene therapy
trial at the University
of Pennsylvania (ScienceNOW, 9 December 1999).
A «cure» for haemophilia is one step closer, following results published in the
New England Journal
of Medcine
of a groundbreaking
gene therapy
trial led by the NHS in London.
The research, part
of a phase I clinical
trial to test the safety
of the treatment, was published as a letter to the editor in The
New England Journal
of Medicine earlier this week and will be in the September issue
of Human
Gene Therapy.
The researchers used the
new technique to mutate the
genes CXCR4 and CCR5, which encode receptor molecules that different strains
of the HIV virus use to sneak in and infect immune cells and which have been targeted in previous cell therapy
trials.
Dr. Srivastava has co-founded a biotechnology company to help find
new cures for many human diseases and one
of the developmental
genes whose role he discovered, Thymosin β4, is currently in clinical
trials for patients suffering ischemic damage to the heart.
CRISPR Therapeutics is pioneering a
new class
of medicines, to conduct the first company - sponsored clinical
trial of a CRISPR
gene - edited therapy.
Yesterday a team
of University
of Pennsylvania researchers — led by Dr Pablo Tebas, Professor Carl June, and Dr Bruce Levine — announced the successful conclusion
of a clinical
trial to evaluate the safety
of a
new gene therapy technique for treating HIV.
The program is unique in its concentration
of nationally and internationally recognized leaders in the areas
of new drug development, cell and
gene therapy and design and conduct
of both local and national clinical
trials through the Children's Oncology Group (COG).
He also runs clinical
trials of new treatments in HD including «
gene silencing» drugs.
Grants were awarded in 6 categories that cover a range
of disciplines: Clinical
trials and outcome measures, Biomarkers
of progression,
Gene studies, Underlying pathology,
New disease models and Rehabilitation
trials.
As CRISPR - Cas9 starts to move into clinical
trials, a
new study published in Nature Methods has found that the
gene - editing technology can introduce hundreds
of unintended mutations into the... Read more
New treatments are being tested to tackle the crippling difficulties
of vertigo, including a clinical
trial of prosthetic ear implants and ear
gene therapy, with initial work revealing novel aspects
of brain anatomy linked to balance which could be used as targets for future treatments.