[1] Other early trials have been used to explore the treatments potential,[2] including for therapeutic use of recombinant adeno-associated virus (rAAV) vectors.
This gene contains 50 exons with a coding region spanning 6.7 kb and thus requires a viral vector capable of handling such a relatively large insert.
[3] These TLRs can initiate downstream effects that can eventually result in the loss of the infected cell and complications in the treated patient.
Other work by researchers suggests interferon may play an important role in preventing successful infection of the target cell.
[3] Although these immune system responses may present hurdles to future medical treatments, researchers may manage the issue with different methods.
Lentiviral vectors may offer substantial promise for the treatment of many genetic disorders manifesting themselves in the retina, such as LCA-2 and Stargardt disease.
Two copies of a nonfunctional ABCA4 gene result in a buildup of a retinoid compound known as A2E, which is believed to act like a detergent inside cells, causing massive cellular damage.
[4] As A2E buildup from the photoreceptor cells collects in the retinal pigment epithelium, severe visual loss occurs.
When researchers treated Stargardt disease-affected mice with a lentiviral vector containing a functional ABCA4 gene, A2E buildup in the retinal pigment epithelium decreased.