Applications of gene modification technologies in the treatment of inherited diseases
Introduction and Purpose: In last years gene modification technologies such as CRISPR/Cas9 has had a revolutionary impact on the treatment of inherited diseases. Technologies developed from bacterial defense mechanisms, has become a basic tools in scientific research and medical therapies. In our a...
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| Format: | Article |
| Language: | English |
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Kazimierz Wielki University
2024-04-01
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| Series: | Journal of Education, Health and Sport |
| Subjects: | |
| Online Access: | https://apcz.umk.pl/JEHS/article/view/50073 |
| _version_ | 1797201149898522624 |
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| author | Maciej Superson Katarzyna Szmyt Klaudia Wilk-Trytko Julia Krasnoborska Sylwia Samojedny Katarzyna Szymańska Kamil Walczak |
| author_facet | Maciej Superson Katarzyna Szmyt Klaudia Wilk-Trytko Julia Krasnoborska Sylwia Samojedny Katarzyna Szymańska Kamil Walczak |
| author_sort | Maciej Superson |
| collection | DOAJ |
| description |
Introduction and Purpose: In last years gene modification technologies such as CRISPR/Cas9 has had a revolutionary impact on the treatment of inherited diseases. Technologies developed from bacterial defense mechanisms, has become a basic tools in scientific research and medical therapies. In our article we provided an overview of applications of gene modifications technologies, directly focusing on CRISPR/Cas9, in genetic disease treatment.
State of Knowledge: New applications of CRISPR/Cas9 are still being explored. Treating inherited diseases such as cystic fibrosis, Duchenne muscular dystrophy, thalassemia, hemophilia, Huntington's disease, Crigler-Najjar syndrome, sickle cell anemia, Marfan syndrome, and phenylketonuria, is feasible with this novel technique. A comparative analysis with other gene editing methods highlights CRISPR/Cas9's efficacy, ease of use, and multiplexing capabilities.
Summary: CRISPR/Cas9 is a groundbreaking technology with broad applications in genetic research and therapy. Its ease of use, cost-effectiveness, and ability to target multiple genes simultaneously position it as a preferred method. However, there are some challenges associated with precision issues and ethical considerations in human embryo gene editing. As CRISPR/Cas9 continues to evolve, responsible application and ethical considerations are important for maximizing its potential in treatment of genetic diseases.
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| first_indexed | 2024-04-24T07:42:58Z |
| format | Article |
| id | doaj.art-ef58789faf3549999ce0b79330448f60 |
| institution | Directory Open Access Journal |
| issn | 2391-8306 |
| language | English |
| last_indexed | 2024-04-24T07:42:58Z |
| publishDate | 2024-04-01 |
| publisher | Kazimierz Wielki University |
| record_format | Article |
| series | Journal of Education, Health and Sport |
| spelling | doaj.art-ef58789faf3549999ce0b79330448f602024-04-19T10:54:06ZengKazimierz Wielki UniversityJournal of Education, Health and Sport2391-83062024-04-016610.12775/JEHS.2024.66.002Applications of gene modification technologies in the treatment of inherited diseasesMaciej Superson0Katarzyna Szmyt1Klaudia Wilk-Trytko2Julia Krasnoborska3Sylwia Samojedny4Katarzyna Szymańska5Kamil Walczak6University Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów PolandUniversity Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów PolandUniversity Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów PolandMedicadent Clinic, Piątkowska 110a, 60-649, Poznań, PolandUniversity Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów PolandUniversity Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów PolandUniversity Clinical Hospital Fryderyk Chopin, ul. Fryderyka Szopena 2, 35-055 Rzeszów Poland Introduction and Purpose: In last years gene modification technologies such as CRISPR/Cas9 has had a revolutionary impact on the treatment of inherited diseases. Technologies developed from bacterial defense mechanisms, has become a basic tools in scientific research and medical therapies. In our article we provided an overview of applications of gene modifications technologies, directly focusing on CRISPR/Cas9, in genetic disease treatment. State of Knowledge: New applications of CRISPR/Cas9 are still being explored. Treating inherited diseases such as cystic fibrosis, Duchenne muscular dystrophy, thalassemia, hemophilia, Huntington's disease, Crigler-Najjar syndrome, sickle cell anemia, Marfan syndrome, and phenylketonuria, is feasible with this novel technique. A comparative analysis with other gene editing methods highlights CRISPR/Cas9's efficacy, ease of use, and multiplexing capabilities. Summary: CRISPR/Cas9 is a groundbreaking technology with broad applications in genetic research and therapy. Its ease of use, cost-effectiveness, and ability to target multiple genes simultaneously position it as a preferred method. However, there are some challenges associated with precision issues and ethical considerations in human embryo gene editing. As CRISPR/Cas9 continues to evolve, responsible application and ethical considerations are important for maximizing its potential in treatment of genetic diseases. https://apcz.umk.pl/JEHS/article/view/50073Crispr/cas9gene modificationgene therapygenetic diseases |
| spellingShingle | Maciej Superson Katarzyna Szmyt Klaudia Wilk-Trytko Julia Krasnoborska Sylwia Samojedny Katarzyna Szymańska Kamil Walczak Applications of gene modification technologies in the treatment of inherited diseases Journal of Education, Health and Sport Crispr/cas9 gene modification gene therapy genetic diseases |
| title | Applications of gene modification technologies in the treatment of inherited diseases |
| title_full | Applications of gene modification technologies in the treatment of inherited diseases |
| title_fullStr | Applications of gene modification technologies in the treatment of inherited diseases |
| title_full_unstemmed | Applications of gene modification technologies in the treatment of inherited diseases |
| title_short | Applications of gene modification technologies in the treatment of inherited diseases |
| title_sort | applications of gene modification technologies in the treatment of inherited diseases |
| topic | Crispr/cas9 gene modification gene therapy genetic diseases |
| url | https://apcz.umk.pl/JEHS/article/view/50073 |
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