AVANÇOS DA TERAPIA GÊNICA –TÉCNICAS UTILIZADAS PARA MANIPULAÇÃO GENÉTICA

• DOI: 10.22533/at.ed.6241923083

• Palavras-chave: Terapia gênica. CRISPRCas9, iPSC, Bioética

• Keywords: Gene therapy. CRISPR-Cas9, iPSC, Bioethics

• Abstract:

  Gene therapy is a procedure that

  involves the genetic modification of cells as a

  way to treat diseases. Genes influence virtually

  all human diseases, either by encoding abnormal

  proteins directly responsible for the disease, or

  by determining susceptibility to environmental

  agents that induce it. Two important techniques

  are studied in this work. The CRISPR-Cas9

  system and the creation of induced pluripotent

  stem cells (iPSC). The CRISPR-Cas9 system

  (Clustered Regularly Interspaced Short

  Palindromic Repeats) has been outstanding due

  to its simplicity of handling in the laboratory and

  to allow the correction of genetic defects. The

  other technique aims at the use of hematopoietic

  stem cells, which have become an ideal target

  for gene transfer. An example of this combination of gene therapy and stem cells would

  be the generation of gene transfer vectors for the creation of induced pluripotent stem

  cells (iPSC). Bioethics is always present to evaluate the risks of the procedures and the

  moral implications involved. The therapy is still experimental, but it has great potential

  and should have a great impact on all aspects of medicine. In this way, the objective

  of this work was to approach the gene therapy, its limitations, applications and future

  perspectives through literature review.

• Número de páginas: 15