Despite the marginal improvements in survival of patients suffering from malignant glioma
treated with gene therapy vectors, the clinical trials conducted so far using viral vectors, in particular adenoviral vectors, have proven that the use of adenoviral vectors is a safe therapeutic approach, even in large, multicenter, phase 3 clinical trials.
«In the group that was not
treated with the gene therapy, the majority of the animals die from aplastic anaemia, and they also die much sooner.»
Plaques decreased substantially in mice
treated with gene therapy to increase activity of the enzyme neuraminidase 1 (NEU1) in a region of the brain involved in learning and memory.
Researchers found that all 15 children with SMA1
treated with the gene therapy were functioning without any major disability or progression of their disease.
«The T cell leukaemia seen in one of the patients
treated with gene therapy for SCID - X1 is extremely regrettable and our sympathies are with the child and his family as he undergoes chemotherapy.
2/12/2008 Gene Therapy Protocol at UCSD Activates Immune System in Patients with Leukemia A research team at the Rebecca and John Moores UCSD Cancer Center at University of California, San Diego (UCSD) reports that patients with chronic lymphocytic leukemia (CLL) who were
treated with a gene therapy protocol began making antibodies that r...
Two children
treated with gene - edited cells to kill their cancers are both doing well more than a year later.
Before he was
treated with the gene therapy, Jake would wake up early before work to inject three times a week as well as injecting whenever he had an injury to stop the bleeding.
Once
treated with the gene that increased new neuron population, however, the mice reverted to assessing their new surroundings first, as usual.
Five children with a genetic disease that wipes out their immune system have successfully been
treated with gene therapy
SCID was the first condition to be
treated with gene therapy more than 20 years ago.
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.
«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.»
One blind group was
treated with the gene therapy, while the other two groups were not.
They found that meadow voles
treated with gene therapy acted more like their prairie vole counterparts — they spent more time huddling near their original companion.
Although the researchers don't know precisely how the chaperone molecule works, the difference was unmistakable: Less than 40 % of cells
treated with gene therapy had clumps, compared to 70 % of controls.
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.
When the researchers paired female mice
treated with the gene therapy with males, the females were still able to become pregnant — and have healthy babies — within the first six weeks, because of those follicles that had already started growing in the ovaries.
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.
Although there has been some recent success in
treating disease
with gene therapy, these types of procedures are far from perfect and can put a user's health — and life — at risk.
The first
gene therapies approved in the United States are
treating patients
with certain types of leukemia and lymphoma.
These findings allowed researchers to create a chimera virus: a mouse virus
with a human viral
gene that can be used to test molecules that inhibit human LANA protein in an animal model of disease,
treating not only human herpes virus infection but also its associated cancers.
Specifically, they have found unnaturally high levels of antibiotic resistance
genes in sediments where the river comes into contact
with treated municipal wastewater effluent and farm irrigation runoff as it flows 126 miles from Rocky Mountain National Park through Fort Collins and across Colorado's eastern plain, home to some of the country's most densely packed livestock operations.
EXTREME differences in the way
genes are expressed by fetuses
with Down's syndrome could point to ways to
treat the condition in the womb.
Building the knowledge base requires humans to teach computers key concepts from curated articles;
with modest online training, anyone who reads English can scan research papers for key terms — names of
genes, proteins, diseases, and drugs — and use online marking tools to document relationships between them (for example, drug X
treats disease Y).
The survey, described today in a Policy Forum published by Science, randomly presented people
with different vignettes that described genome editing being used in germline or somatic cells to either
treat disease or enhance a human
with, say, a
gene linked to higher IQ or eye color.
The team found that humans are equipped
with tiny differences in a particular regulator of
gene activity, dubbed HARE5, that when introduced into a mouse embryo, led to a 12 % bigger brain than in the embryos
treated with the HARE5 sequence from chimpanzees.
These cells have been notoriously difficult to
treat with previous
gene - delivery techniques.
They are also studying whether the nanoparticles can be used
with the CRISPR / Cas9
gene - editing technique to
treat genetic lesions related to retinal degenerative diseases.
The researchers assessed LPLD - related acute abdominal events that required hospital care in a small group of patients
treated with a single dose of the
gene therapy product Glybera ®.
And in an ironic twist, the work could yield a new way to
treat common ailments such as heart disease and cancer
with gene therapies.
Those
with the
gene are
treated with Herceptin, a drug that we know provides a benefit.
To learn which
genes in the mussel make the byssal threads, the researchers first
treated the threads
with the enzyme pepsin to liquefy their interlocking networks of proteins.
PARP inhibitors are used primarily to
treat tumors linked to defects in BRCA
genes, but may show promise in other cancers when combined
with other drugs.
«Stem cell
gene therapy could be key to
treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of patients
with the deadly disease.»
Between 2008 and 2011 Jean Bennett, a neuroscientist at the University of Pennsylvania, and her colleagues used
gene therapy to
treat blindness in 12 adults and children
with Leber's congenital amaurosis (LCA).
This tells us how these
genes evolved and gives insight on what the
genes do and how mutations might be
treated with drugs.
A baby's leukemia was successfully
treated in 2015
with CAR - T cells engineered
with gene editors known as TALENs.
When the group over-expressed the
gene for MKP - 1 in the brains of healthy rats, the animals began to show signs of depression which disappeared when
treated with an antidepressant (Nature Medicine, DOI: 10.1038 / nm.2219).
In the lab, replacing malfunctioning
genes with working ones has succeeded in curing or
treating animals
with hemophilia, (the animal analogs of) depression, and other afflictions.
Diamond's lab circumvented this problem by creating female mice that had a key interferon
gene knocked out; in a second experiment, they
treated pregnant animals
with an anti-interferon antibody.
2009: A resounding success brings
gene therapy back into the limelight: Jean Bennett and Albert Maguire announce that 12 patients
with a degenerative disorder leading to total blindness were
treated with viruses bearing a replacement
gene, and all regain some vision.
Sensory hair cells in the cochlea of a Beethoven mouse
treated with TMC2
gene therapy.
Global
gene expression studies found that the LPA -
treated mice shared many similar molecular markers as those found in humans
with schizophrenia.
Gene editing could include altering
genes in one person — say to
treat leukemia in one patient or make a cosmetic change — but, more controversially, it could also include making changes to the germ line that would then alter the genome for an individual's children, grandchildren and the following generations,
with potentially unknown repercussions.
Despite much effort, no one has managed to develop an effective form of
gene therapy to
treat the lung problems of people
with cystic fibrosis.
In the study, 50 patients
with BRCA
gene mutations
treated at one of 18 centers took veliparib by mouth twice a day.
Among patients
with non-small cell lung cancer (NSCLC) fueled by ALK
gene alterations who were being
treated with crizotinib (Xalkori), a decrease in the number of circulating tumor cells (CTCs) harboring increased copies of the ALK
gene over the first two months of treatment was associated
with increased progression - free survival.
The researchers
treated 63 cancer cell lines (26 breast, 14 colorectal and 23 ovarian)
with low - dose 5 - azacitidine (AZA), an FDA - approved drug for myelodysplastic syndrome, that reverses epigenetic changes by stripping off the methyl group that silences the
gene.
In 2013 another study will focus on a family in Colombia that carries one of these rogue
genes,
treating them
with the amyloid - fighting drug Crenezumab.