Studies are also underway evaluating therapeutic
gene transfer approaches to treat cardiac and skeletal muscle dysfunction in X-linked muscular dystrophy golden retriever dogs.
This study will combine direct tumor cell killing (TK) and immune - mediated stimulatory (Flt3L)
gene transfer approaches delivered by first generation adenoviral vectors.
However, like most
gene transfer approaches a major concern with ZFN technology is the potential for oncogenesis due to off - target effects.
«One way of treating patients with limb - girdle muscular dystrophy might be to use
a gene transfer approach to treat the muscular dystrophy, and a pharmacological approach to treat the smooth muscle dysfunction,» he said.
Her research initially focused on the identification and characterization of juvenile dilated cardiomyopathy in Portuguese water dogs and has led to several ongoing studies, including most recently the evaluation of various candidate genes for use in a therapeutic
gene transfer approach to improve cardiac function in failing hearts.
Not exact matches
This
approach entails
transferring a healthy (non-mutant) copy of the defective
gene to restore the expression of the missing protein.
In one such
approach, researchers surgically remove brain cells, use viruses to
transfer genes to the cells, and then graft them back into the animal's brain tissue.
His team's
approach is based on
gene therapy, where a «tame» virus is harnessed to
transfer a
gene into target cells in the recipient.
Dr. Stoddart's main research focus is using autologous stem cells and
gene transfer to repair musculoskeletal tissues using a cell therapy
approach.
«
Gene therapy approaches fall into two main categories: (1) the transfer of a gene that stimulates the immune system and (2) the insertion of genes within the tumor that sensitizes the tumor to a relatively nontoxic «prodrug.&ra
Gene therapy
approaches fall into two main categories: (1) the
transfer of a
gene that stimulates the immune system and (2) the insertion of genes within the tumor that sensitizes the tumor to a relatively nontoxic «prodrug.&ra
gene that stimulates the immune system and (2) the insertion of
genes within the tumor that sensitizes the tumor to a relatively nontoxic «prodrug.»
The latest developments in CAR - T translation and manufacturing including alternate
gene transfer methods to deliver CARs; novel
gene editing
approaches, advances in CAR design to improve safety and efficacy, and progress in creating an «off - the - shelf» allogeneic CAR - T therapy.
«The data from this study show the promise of vector - mediated
gene transfer as an
approach for immunization,» says Pat Fast, chief medical officer at the International AIDS Vaccine Initiative.
Ultimately the goal is to translate this work to
gene transfer treatment
approaches for canine idiopathic dilated cardiomyopathy and human heart failure.