Hemophilia is an inherited bleeding disorder caused by the lack of a protein necessary for blood clotting. Gene therapy hemophilia involves introduction healthy gene into patient's cells to produce missing protein. There are two main types hemophilia: ex vivo therapy, which extraction from patient, these in laboratory, and reintroduction modified patient; direct delivery body using viral vector...

objective: the greatest challenge in cancer gene therapy is to achieve the high specificity and efficiency in targeting of cancer cells. because the goal of cancer gene therapy is to eradicate cancer cells, many therapeutic genes could be detrimental if unintentionally expressed in normal cells. using promoter of the genes which are expressed specifically in cancer cells or have much more expre...

Background: Radiation therapy is among the most conventional cancer therapeutic modalities with effective local tumor control. However, due to the development of radio-resistance, tumor recurrence and metastasis often occur following radiation therapy. In recent years, combination of radiotherapy and gene therapy has been suggested to overcome this problem. The aim of the current study was to e...

Objective(s): The use of antisense oligonucleotides (AOs) to restore normal splicing by blocking the recognition of aberrant splice sites by the spliceosome represents an innovative means of potentially controlling certain inherited disorders affected by aberrant splicing. Selection of the appropriate target site is essential in the success of an AO therapy. In this study, in search for a splic...

We present updated results from ASPIRO (NCT03199469), investigating gene replacement therapy with AT132 for XLMTM patients. XLMTM, an ultra-rare, life-threatening myopathy caused by variants in the MTM1 gene, leads to impaired neuromuscular and respiratory function, early death.

The prevalence of familial atrial fibrillation (AF) in the general population and structure AF is considered, genetic predictors pathogenetic mechanisms remodeling are analyzed. assessment risk occurrence, prediction its outcomes effectiveness therapy, as well prospects for gene therapy discussed.

background: the herpes simplex virus (hsv) ul41 gene product, virion host shutoff (vhs) protein, mediates the rapid degradation of both viral and cellular mrna. this ability suggests that vhs protein can be used as a suicide gene in cancer gene therapy applications. the recent reports have shown that the degradation of cellular mrna during herpes simplex infection is selective. rna containing a...

Gene therapy as a modern therapeutic approach has not yet advanced to a globally-approved therapeutic approach. Lack of adequate reliable gene delivery system seems to be one of the major reasons from the pharmaceutical biotechnology point of view. Main obstacles delaying successful application of human gene therapy are presented in this review. The unique advantages of non-biological gene carr...