Możliwości terapii mukowiscydozy. Terapia genowa
Słowa kluczowe:
mukowiscydoza, płuca, PEP, terapia genowa, CRISPR/Cas9Streszczenie
Abstrakt
Mukowiscydoza to nieuleczalna choroba autosomalne recesywna wywołana mutacją genu regulatora transbłonowego CFTR. Choroba obejmuje drogi oddechowe, trzustkę, układ pokarmowy i gruczoły potowe. Najbardziej narażona na tę chorobę jest populacja rasy kaukaskiej. Mediana życia pacjentów ciągle rośnie, dzięki poszerzających się typach terapii. Zaliczamy do nich leczenie farmakologiczne, fizjoterapie, leczenie żywieniowe oraz przeszczep płuc. Szansę na wyleczenie chorych daje terapia genowa, dynamicznie rozwijająca się dziedzina medycyny, która narazie jest w fazie badań klinicznych.
Słowa kluczowe: mukowiscydoza, płuca, PEP, terapia genowa, CRISPR/Cas9.
Abstract
Cystic fibrosis is an incurable autosomal recessive disease caused by a mutation of the CFTR transmembrane regulator gene. The disease involves the respiratory tract, pancreas, digestive system and sweat glands. The most vulnerable is the Caucasian population. The median lifespan continues to grow, thanks to expanding types of treatments. These include pharmacological treatment, physiotherapy, nutrition treatment and lung transplantation. Gene therapy gives a chance to cure the disease, the field of medicine is dynamically developing, which is currently in the phase of clinical trials.
Key words: cystic fibrosis, lungs, PEP, gene therapy, CRISPR/Cas9.
Rozdziały
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Możliwości terapii mukowiscydozy. Terapia genowa
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Opublikowane
1 września 2023
Prawa autorskie (c) 2023 Matylda Kujawińska, Iga Kwas, Kacper Kuzan
Licencja
Utwór dostępny jest na licencji Creative Commons Uznanie autorstwa – Użycie niekomercyjne – Bez utworów zależnych 4.0 Międzynarodowe.
