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.
Not exact matches
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.
Wilson famously led a
gene therapy study
with an adenovirus
vector that killed a patient, Jesse Gelsinger, in 1999 — a major setback for the entire field.
With this in mind, independent teams led by Scott and Herzog took the conventional viral -
vector approach to inducing tolerance through
gene therapy.
This has limited the application of
gene therapy, along
with other concerns in the field, like the safety of the delivery
vector.
With gene therapy the VNA genetic material is delivered to animals by a
vector that induces the animals to produce their own antitoxin VNA proteins over a prolonged period of time, thus preventing illness from toxin exposures.
There's also a long - term risk from the
gene therapy that we think is small, because we've genetically altered the T cells
with a viral
vector that's actually a modified, non-pathogenic form of HIV, the AIDS virus.
In the current work we used adenoviral - type 5 (dE1 / E3)(Cytomegalovirus promoter)
with human ABCA10 transgene (Ad - h - ABCA10) purchased from
Vector Labs ® in order to investigate whether
gene therapy can be used as a pre-treatment to enhance the efficiency of inhaled cisplatin.
With the aim of pioneering a safe, effective
gene therapy approach, St. Jude scientists have spent years developing an innovative
vector for X-linked SCID.
But what's more, the transplanted cells keep producing a foreign protein slipped inside
with the help of a lentiviral
vector, the kind usually used for
gene therapy.
Since lentiviral
vectors can deliver a large amount of DNA (~ 8 kb)
with a relatively low immune response, it should come as no surprise that researchers are interested in developing these
vectors for
gene therapy.
AAV
gene therapy vectors are emerging as the
gene transfer vehicle
with high potential for use in the CNS as they transduce post mitotic cells that mediate the sustained, long term
gene expression required in chronic progressive diseases.