In addition to its emphasis on producing vaccines, the lab specializes
in gene and cell therapies, including stem cell products.
GLP Test Facilities combine skills and expertise
in gene and cell therapy research, pharmaceutical research and development, pathology and quality assurance.
ASGCT is the primary membership organization for scientists, physicians, professionals, and patient advocates involved
in gene and cell therapy.
ASGCT's annual meeting is the essential scientific conference for professionals
in gene and cell therapy.
ASGCT is your unbiased source for reliable, scientific information, news, and breakthroughs
in gene and cell therapy.
Not exact matches
Birds, for example, can regenerate hair
cells —
and lately researchers have found a few similar effects
in mammals that used
gene and drug
therapy.
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.
• Vineti, a San Francisco - based
cell and gene therapy software
and analytics company, raised $ 13.75 million
in Series A funding.
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.
With major clinical successes
in areas such as CAR - T,
gene therapy, immune - oncology,
cell therapy and gene editing, many see 2017 as the year that biotech really came of age.
Risk Versus Reward: The Value of
Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
Therapy for Patients
and Investors Source: Streetwise Reports (4/25/18) The
cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
therapy space, encompassing disruptive new treatment including stem
cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developm
cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage devel
therapy, immunotherapy
and gene editing, has begun to mature, with a handful of product approvals
and others
in late - stage development.
The
cell therapy space, encompassing disruptive new treatment including stem
cell therapy, immunotherapy
and gene editing, has begun to mature, with a handful of product approvals
and others
in late - stage development.
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.
In the study, they found that miR - 182 suppressed Bcl2L12, a cancer gene that blocks cancer cell death in response to chemo - and radiation therap
In the study, they found that miR - 182 suppressed Bcl2L12, a cancer
gene that blocks cancer
cell death
in response to chemo - and radiation therap
in response to chemo -
and radiation
therapy.
Because of this, families
and doctors often shrink from transplant options, particularly when it comes to treating children,
and it will limit the extent to which the breakthroughs
in gene therapy and gene editing will be applied, explained Scadden, who is a practicing hematologist at MGH
and chairman of Harvard's Department of Stem
Cell and Regenerative Biology.
The stem
cell gene therapy could be applicable for 60 percent of people with Duchenne, which affects approximately 1
in 5,000 boys
in the U.S.
and is the most common fatal childhood genetic disease.
These include the ability to bring new, innovative products to the market; progress
in oncology, such as the approval of Genentech's drug Avastin for breast cancer
and advances
in the use of
gene therapy, despite some setbacks; continuing progress
in research on stem
cells; the emergence of treatments for previously untreated diseases;
and solutions for food
and fuel shortages, such as biocrops
and biofuels.
Two of 10 children treated with
gene therapy for SCID
in a French trial develop leukemia, researchers announced
in 2002,
and it is discovered that the virus had inserted
genes in several unexpected places around the genome, leading the
cells to become cancerous.
Dwarki
and Jaime Escobedo improved the AAV's ability to insert
genes into chromosomes by adding a
gene promoter region from cytomegalovirus, known to be active
in the target for their
gene therapy, muscle
cells.
Epigenetic
therapies are thought to work
in two ways to fix these errors
in cancer
cells — by correcting the «position» of the
gene switches
and by making the
cell appear as though it's infected by a virus, triggering the immune system.
In a last - ditch effort to save a dying 7 - year - old boy, scientists have used stem
cells and gene therapy to replace about 80 percent of his skin.
Muscle biologists Qi Long Lu
and Terence Partridge at the Medical Research Council Clinical Sciences Centre
in London, U.K.,
and their colleagues decided to combined the antisense strategy with a chemical often used
in gene therapy because it is known to improve delivery of DNA into
cells.
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.
Researchers at the Center for
Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital
and Houston Methodist have developed an alternative treatment
in which virus - specific
cells protect patients against severe, drug - resistant viral infections.
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 brainste
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 brainste
in the auditory portion of the brainstem.
Delivering a single injection of a scar - busting
gene therapy to the spinal cord of rats following injury promotes the survival of nerve
cells and improves hind limb function within weeks, according to a study published April 2
in The Journal of Neuroscience.
It showed effectiveness against liver cancer
in a phase II clinical trial
and will move into a phase III trial later this year, David Kirn, an oncologist
and the company's president
and chief executive officer, said at a recent meeting of the American Society for
Gene &
Cell Therapy in Washington, D.C..
«Single
gene encourages growth of intestinal stem
cells, supporting «niche»
cells,
and cancer: Finding
in mice could lead to new
therapies for damaged organs, cancer.»
At the Center for
Cell and Gene Therapy at Baylor College of Medicine
in Houston, director Malcolm Brenner, a geneticist, says he has turned «AdV from poacher into gamekeeper.»
Still, by identifying the human hairless
gene as an important master switch
in regulating
cell death
in a hair follicle — a discovery that could lead to
gene therapies for unwanted hair growth — Christiano emerged as a new star
in the field,
and a glamorous one.
The technical advances emerging from
gene therapy have fueled the larger fields of
cell and immune
therapy, where DNA, immune molecules,
and viruses are all elements to be manipulated,
in concert or one at a time.
In an effort to expand the number of cancer
gene mutations that can be specifically targeted with personalized
therapies, researchers at University of California San Diego School of Medicine
and Moores Cancer Center looked for combinations of mutated
genes and drugs that together kill cancer
cells.
The proposed clinical trial,
in which researchers would use CRISPR to engineer immune
cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety
and ethics of
gene therapy trials funded by the U.S. government
and others.
«Research into basic workings of immune system points to way of improving
therapies for cancer: Differences
in wiring of «exhausted»
and effective T
cells indicate possible
gene - editing targets.»
In a step toward accelerating the production of new gene therapies, scientists report in ACS Nano that they have developed remote - controlled, needle - like nanospears capable of piercing membrane walls and delivering DNA into selected cell
In a step toward accelerating the production of new
gene therapies, scientists report
in ACS Nano that they have developed remote - controlled, needle - like nanospears capable of piercing membrane walls and delivering DNA into selected cell
in ACS Nano that they have developed remote - controlled, needle - like nanospears capable of piercing membrane walls
and delivering DNA into selected
cells.
Although the
gene /
cell therapy strategy was highly successful
in laboratory mice, the authors stressed that additional research
and testing are needed before the
therapy could be tested
in humans.
«Identifying targets essential to
cell survival
in tumor suppressor
genes has long been an investigational goal with the aim of offering cancer - specific vulnerabilities for targeted
therapy,» said Ronald DePinho, M.D., professor of Cancer Biology, MD Anderson president,
and senior author for the Nature paper.
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.
One potential treatment for CF is
gene therapy,
and a major challenge
in gene therapy is packaging replacement
genes so they can be delivered to the target
cells.
By performing a genome - wide screen
in breast cancer
cells, Dr. Oesterreich
and her colleagues identified a
gene called HOXC10 as one that the cancer seems to modify to allow continued tumor growth
in patients whose cancer becomes resistant to traditional
therapies.
Dr. Levine directs the Clinical
Cell and Vaccine Production Facility (CVPF), which develops, manufactures, and tests novel cell and gene therapies in clinical trials at Penn and collaborating instituti
Cell and Vaccine Production Facility (CVPF), which develops, manufactures,
and tests novel
cell and gene therapies in clinical trials at Penn and collaborating instituti
cell and gene therapies in clinical trials at Penn
and collaborating institutions.
A new
gene therapy treatment has restored some sight
in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome
in which, because of a broken or missing
gene called RPE65, retinal photoreceptor
cells malfunction
and eventually die.
A year later, researchers corrected the faulty
gene in lab - grown lung
cells and felt it wouldn't be long before
gene therapy could be used to implant correct versions of the
gene into people with CF.
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.
Under the proposed law,
gene therapy will be approved only for the treatment of people with genetic diseases such as cystic fibrosis
and will not be allowed
in germ
cells, where genetic alterations would be passed on to the next generation.
The specificity of this DNA cutting activity has made CRISPR - Cas the darling of
gene therapy researchers, who have modified it to make precise changes
in the genomes of cultured
cells, laboratory animals,
and even humans.
In this way we hope to lay a clinical groundwork for two new techniques: liver
cell transplantation
and liver
gene therapy.»
For the animal experiments, Savio Woo of the Center for
Gene Therapy at Baylor College of Medicine
in Houston
and his colleagues first isolated liver
cells from transgenic mice that produce the human protein a1 - antitrypsin
in their livers, from where it is secreted into the blood.
The research team found that when a particular mitochondrial fission protein (GTPase dynamin - related protein - 1 — Drp1) was blocked using either
gene -
therapy or a chemical approach
in experimental models of PD
in mice, it reduced both
cell death
and the deficits
in dopamine release — effectively reversing the PD process.