Gene Therapy Approaches

Non-viral approaches to gene therapy.

There have been rapid advances in the development of non-viral techniques for gene transfer. Although viruses are highly evolved to infect mammalian cells, they have several limitations. The general theme is to mimic the advantageous components of viral systems whilst separating them from their limiting functions. The systems described here are broadly divided into true non-viral techniques and...

Gene therapy approaches for bone regeneration.

Gene therapy represents a promising approach for delivering regenerative molecules to specific tissues including bone. Several laboratories have shown that virus-based BMP expression vectors can stimulate osteoblast differentiation and bone formation in vivo. Both in vivo and ex vivo transduction of cells can induce bone formation at ectopic and orthotopic sites. Adenovirus and direct DNA deliv...

ژن درمانی Gene Therapy

Cell- and gene-therapy approaches to inner ear repair.

Sensorineural hearing loss is the most common sensory disorder in humans. It is primarily due to the degeneration of highly specialised mechanosensory cells in the cochlea, the so-called hair cells. Hearing problems can also be caused or further aggravated by the death of auditory sensory neurons that convey the information from the hair cells to the brain stem. Despite the discovery of stem/pr...

Gene therapy approaches to the treatment of hemoglobinopathies Review

Hemoglobinopathies such as thalassemia and sickle cell anemia are potentially amenable to gene therapy. Applicable gene therapy strategies can be divided into four categories: those that replace the faulty gene with a complete transcript ional unit , those that activate transcription of fetal hemoglobin genes, those that modify the endogenous gene itself , and those that attempt to repair the d...