Research on Genetic Diseases
Today, November 10, 2020, we celebrate Science Day.
We want to make special mention of the evolution in genetic treatments.
Evolution in science
Genetic therapy uses genetic material to treat diseases of this group, which, as we already know, Alpha 1 Antitrypsin Deficiency is part of it.
This therapy tries to modulate cell function, being able to correct the deficiency caused by the loss or alteration of a gene, by modifying the expression of proteins.
Thus, protein coding sequences, antisense probes, triple DNA and ribozomes can be considered the drugs of this treatment.
The complexity of this resides in the set of techniques for the distribution of these “drugs”, that is, the transfer of this material to the target cells.
Genetic alterations beneficial to health
Thanks to the advance in the knowledge about genetics, mechanisms that regulate cell division and differentiation have been discovered.
The development of technology has helped to understand the recombination of DNA to amplify the biological response.
Through the identification, isolation and sequencing of functional genes.
The reason for the healthier outlook for gene therapy in 2020 is “improvements in gene transfer technology.”
In the case that concerns us, with respect to respiratory patients with Alpha 1 deficiency, trials have shown some unusual tolerogenic properties.
The study shows that patients develop regulatory T cells against AAV coat proteins that prevent them from reacting negatively, even without pharmaceutical immunosuppression. “
We want to continue celebrating Science Day, thanking all professionals.
Especially to health workers and researchers, who ensure the progress and improvement of the quality of life of our patients.