One can envision
similar gene therapy applications.
Both De Luca and Marinkovich's teams are exploring
a similar gene therapy for another major form of the disease, called dystrophic EB, caused by a different genetic error affecting a larger protein.
The finding could lead to studies of
a similar gene therapy to treat people with inherited blood disorders.
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.
He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a clinical trial to test a
similar gene -
therapy treatment for Parkinson's disease.
The finding suggests that other AAVs used for a
gene therapy targeting the brain or spinal cord might be improved by having the same or a
similar set of amino acids.
But it's still unknown whether the DNA will integrate into chromosomes, says John Polo, a molecular biologist at Chiron Technologies Center for
Gene Therapy in San Diego, California, which is developing
similar vaccines for HIV and the herpes virus.
To prevent this decline in Area X, White's research team used methods
similar to human
gene therapy to insert a version of FoxP2 in male zebra finches.
As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a
similar approach using
gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.
Overall, the
gene therapy was well tolerated and patients in the treatment and placebo groups experienced
similar rates of adverse events.
The team envisions that in the future, deaf people might benefit from
gene therapy similar to the approaches currently being tested in clinical trials for other diseases.
It uses a virus already approved by the Food & Drug Administration for other genetic
therapies in the eye; it delivers an ion channel
gene similar to one normally found in humans, unlike others that employ
genes from other species; and it can easily be reversed or adjusted by supplying new chemical photoswitches.
It's very
similar to
gene therapy in medicine.
Humans have an ortholog of the murine Nrk
gene, and considering that the
gene expression pattern in breast tumor in Nrk mutant mice was
similar to that in human luminal B breast cancer, the findings of this study may lead to further understanding of the mechanisms of human breast cancer suppression and to advances in its diagnosis and
therapy.
Similar to the problem of traditional
gene therapy hitting the wrong spot, ZFNs could make cuts in places where they weren't supposed to and turn on a cancer
gene.
Our definition is
similar to the European Medicines Agency (EMA) definition of Advanced
Therapy Medicinal Product (ATMP): «Medicinal product for human use that is a gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA AT
Therapy Medicinal Product (ATMP): «Medicinal product for human use that is a
gene therapy medicinal product, a somatic cell therapy medicinal product or tissue engineered product» (EMA AT
therapy medicinal product, a somatic cell
therapy medicinal product or tissue engineered product» (EMA AT
therapy medicinal product or tissue engineered product» (EMA ATMP Reg.
«Because
gene therapy may carry certain risks, we set out to see if we could perform a
similar transformation with small molecules, thereby eliminating — or at least reducing — our reliance on genetic manipulation,» says Dr. Ding.
And doctors at the Great Ormond Street Hospital in London recently reported using a
similar gene - editing technique called TALENs, which also recognizes and cuts precise DNA sequences, to engineer immune cells for a
therapy that may have cured two infants of leukemia.
In the article describing the work in Molecular
Therapy, co-authors Charis Himeda, Takako Jones and Peter Jones highlight the important implications for
similar types of genetic diseases: «With increasing evidence that the repeat genome (comprising nearly half the human genome) plays important roles in
gene regulation, additional diseases will likely be found associated with aberrant repetitive genomic sequences,» they write.
Theoretically, this is
similar to how
gene therapy is used to treat cancer, but with important improvements.
The biotech world is avidly exploring the potency of
gene therapy, and Nelles figures that RNA
therapies might offer
similar potential.
PPMD recently hosted a webinar focused on understanding the different
therapies that are being developed that are commonly referred to as
gene therapy, including micro-dystrophin and CRISPR / Cas9, how are they
similar and different, and what are the challenges and limitations for each of them.
Characterization of genetic mutations, genetic testing and development of
gene - based
therapies for muscular dystrophies and inherited metabolic brain diseases
similar to Tay - Sachs disease.