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ATMP Application in FemTech Health
FemTech solutions include treatment for prenatal and perinatal diseases that can affect fetuses due to congenital diseases or genetic disorders.
Among the advanced therapies, gene therapy and genomic editing are included in FemTech products. These advanced therapies offer a treatment for genetic disorders, delivering genes to cells and tissues in prenatal and perinatal life or correcting the mutation of interest directly on the DNA, with innovative techniques such as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) and all of its evolutions.
Among the most interesting objectives of these therapies are fetal stem cells: the genetic correction is directed to the progenitor stem cells, from which all the others originate; in this way the therapeutic gene is transmitted to all subsequent generations of cells, correcting the defect at the base.
Targeting Genetic Disorders Diseases'
The applications of gene therapy and genome editing in the prenatal and perinatal stages of life are constantly evolving.
Recent studies have yielded interesting results for hemophilia B, retinal blindness and X-linked severe combined immunodeficiency (SCID), but the list of candidate diseases continues to grow as our understanding of their respective pathogenesis at the molecular level increases.
The most suitable candidates for the application of gene therapy in utero may have well-characterized monogenic (meaning caused by a single gene) abnormalities causing severe prenatal or perinatal morbidity. Other disorders include those for which an accurate molecular diagnosis can be achieved before birth and those for which there is no effective postnatal therapy [1].
Delivery Platforms for In-Utero Gene Therapy
The primary obstacles to tackle to be able to exploit the enormous potential of gene therapy in utero are biocompatibility and the ability to transport transgenes into cells, as they are unable to pass directly through the cell membrane, and therefore, vectors are needed to reach their goal.
Most gene therapies use viral delivery platforms, characterized by high transduction efficiency. The four main types of viruses used for this purpose are: lentiviruses, retroviruses, adenoviruses and adeno-associated viruses.
Although the use of viral vectors is promising for their efficacy, concerns exist due to their mutagenicity and immunogenicity.
As a result, non-viral vectors have been developed as safer alternatives for delivery of both gene therapy and gene editing in prenatal and perinatal phases. The four principal classes of non-viral delivery platforms are: polymer-based and lipid-based nanoparticles, inorganic nanoparticles and physical methods.
References
1. Peddi NC, Marasandra Ramesh H, Gude SS, Gude SS, Vuppalapati S. Intrauterine Fetal Gene Therapy: Is That the Future and Is That Future Now? Cureus. 2022 Feb 23;14(2):e22521. doi: 10.7759/cureus.22521. PMID: 35371822; PMCID: PMC8951626.
