Öz
Background:
Gene therapy is applied to regulate the functions of mutated or disease-causing genes in human cells and targets nucleotides. Recent years have seen an increasing number of publications reporting successful results from gene therapies, suggesting an increasing scientific curiosity among clinicians.
Methods:
Gene Therapy Symposiums were hosted by the [blinded for review]. The participants answered pre-post-tests, and satisfaction scales.
Results:
A total of 192 participants underwent training, none of whom had previously undergone gene therapy training. Of the sample, 71.9% were female and the median age of the participants was 32 years. Of the total, 84.9% were studying medicine and 15.1% were studying in non-medical fields. Among those studying medicine, 17.2% were students, 18.4% were main specialty research assistants, 38.0% were subspecialty research assistants, 17.2% were specialists and 9.2% were assistant professors/professors. An analysis of the completed pretests revealed that 32.3% had answered the questions correctly, while 58.6% answered the questions correctly after undergoing training. The overall satisfaction score was 91.8 out of 100.
Conclusions:
The results of the pretest revealed the knowledge and awareness of gene therapy among the participants to be low, indicating a need for education programs addressing the subject. Gene therapy has moved beyond the theoretical realm in recent years and is today seeing practical applications. There is an urgent need to train the clinicians and other operatives required for the provision of gene therapies and to develop strategies for tertiary care centers in this field over the next 10 years.
Anahtar Kelimeler
education future gene therapy inherited metabolic disorders spinal muscular atrophy
Kaynakça
- 1. Baltimore D. RNA-dependent DNA polymerase in virions of RNA tumour viruses. Nature. Jun 27 1970;226(5252):1209-11. doi:10.1038/2261209a0
- 2. Jackson DA, Symons RH, Berg P. Biochemical method for inserting new genetic information into DNA of Simian Virus 40: circular SV40 DNA molecules containing lambda phage genes and the galactose operon of Escherichia coli. Proc Natl Acad Sci U S A. Oct 1972;69(10):2904-9. doi:10.1073/pnas.69.10.2904
- 3. Lobban PE, Kaiser AD. Enzymatic end-to end joining of DNA molecules. J Mol Biol. Aug 15 1973;78(3):453-71. doi:10.1016/0022-2836(73)90468-3
- 4. Wade N. Gene therapy caught in more entanglements. Science. Apr 3 1981;212(4490):24-5. doi:10.1126/science.6259731
- 5. Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science. Oct 20 1995;270(5235):475-80. doi:10.1126/science.270.5235.475
- 6. Smith CIE, Blomberg P. [Gene therapy - from idea to reality]. Lakartidningen. Dec 19 2017;114Genterapi - fran ide till verklighet - Annu har fa patienter behandlats och preparaten ar ofta mycket dyra - men utvecklingen gar fort nu.
- 7. Smith KR. Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline. Int J Med Sci. 2004;1(2):76-91. doi:10.7150/ijms.1.76
- 8. https://pubmed.ncbi.nlm.nih.gov/ 9. Arabi F, Mansouri V, Ahmadbeigi N. Gene therapy clinical trials, where do we go? An overview. Biomed Pharmacother. Sep 2022;153:113324. doi:10.1016/j.biopha.2022.113324
- 10. https://clinicaltrials.gov/.
- 11. Ogbonmide T, Rathore R, Rangrej SB, et al. Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). Cureus. Mar 2023;15(3):e36197. doi:10.7759/cureus.36197
- 12. Eminoglu FT, Koc Yekeduz M, Dogulu N, et al. Inherited metabolic disorders in the neonatal intensive care unit: Red flags to look out for. Pediatr Int. Jan 2022;64(1):e14953. doi:10.1111/ped.14953
- 13. Sugarman EA, Nagan N, Zhu H, et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. Jan 2012;20(1):27-32. doi:10.1038/ejhg.2011.134
- 14. T.R. Ministry of Health 2021a. Premarital Spinal Muscular Atrophy (SMA) Carrier Screening Program. T.R. Ministry of Health. General Directorate of Public Health
- 15. T.R. Ministry of Health 2022. Newborn Metabolic and Endocrine Disease Screening Program. T.R. Ministry of Health. General Directorate of Public Health.
- 16. Waldrop MA, Kolb SJ. Current Treatment Options in Neurology-SMA Therapeutics. Curr Treat Options Neurol. Apr 29 2019;21(6):25. doi:10.1007/s11940-019-0568-z
- 17. Schuster DJ, Dykstra JA, Riedl MS, et al. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front Neuroanat. 2014;8:42. doi:10.3389/fnana.2014.00042
- 18. Rashnonejad A, Durmaz B, Özkınay F. The principles of gene therapy and recent advances. Ege Journal of Medicine 2014;53(4):231-240.
Öz
Giriş:
Gen terapisi, insan hücrelerinde mutasyona uğramış veya hastalığa neden olan genlerin fonksiyonlarını düzenlemek için uygulanan ve nükleotidleri hedef alan bir tedavi yöntemidir. Son yıllarda gen terapisi alanında başarılı sonuçlar bildiren yayınların sayısı artmış, bu da klinisyenler arasında bilimsel merakın artışına sebep olmuştur.
Metod:
Gen Terapisi Sempozyumları [blinded for review] ev sahipliğinde düzenlendi. Katılımcılar ön-son testleri ve memnuniyet ölçeklerini yanıtladılar.
Bulgular:
Hiçbiri daha önce gen terapisi eğitimi almamış olan toplam 192 katılımcıya eğitim verildi. Katılımcıların %71,9'u kadındı ve katılımcıların ortanca yaşı 32 yıldı. Toplamın %84,9'u tıp, %15,1'i ise tıp dışı alanlardandı. Tıp alanından katılımcıların %17,2'si öğrenci, %18,4'ü ana uzmanlık araştırma görevlisi, %38,0'ı yan dal araştırma görevlisi, %17,2'si uzman ve %9,2'si doçent/profesördü. Katılımcıların ön test analizinde soruların %32,3’ünü doğru yanıtladığı, eğitim sonrasında ise bu oranın %58,6’ya yükseldiği saptandı. Genel memnuniyet puanı 100 üzerinden 91,8’di.
Sonuçlar:
Ön test sonuçları, katılımcılar arasında gen terapisine ilişkin bilgi ve farkındalık düzeyinin düşük olduğunu ortaya koymuştur ve bu da konuya yönelik eğitim programlarına ihtiyaç duyulduğunu göstermektedir. Gen terapisi son yıllarda teorik alanın ötesine geçmiştir ve bugün pratik uygulamalar görülmektedir. Önümüzdeki 10 yıl içinde gen tedavilerinin sağlanması için gerekli olan klinisyenlerin ve diğer uygulayıcıların eğitilmesine ve üçüncü basamak sağlık merkezleri için bu alanda stratejiler geliştirilmesine acil ihtiyaç vardır.
Anahtar Kelimeler
eğitim gelecek gen tedavisi kalıtsal metabolik hastalık spinal muskuler atrofi
Kaynakça
- 1. Baltimore D. RNA-dependent DNA polymerase in virions of RNA tumour viruses. Nature. Jun 27 1970;226(5252):1209-11. doi:10.1038/2261209a0
- 2. Jackson DA, Symons RH, Berg P. Biochemical method for inserting new genetic information into DNA of Simian Virus 40: circular SV40 DNA molecules containing lambda phage genes and the galactose operon of Escherichia coli. Proc Natl Acad Sci U S A. Oct 1972;69(10):2904-9. doi:10.1073/pnas.69.10.2904
- 3. Lobban PE, Kaiser AD. Enzymatic end-to end joining of DNA molecules. J Mol Biol. Aug 15 1973;78(3):453-71. doi:10.1016/0022-2836(73)90468-3
- 4. Wade N. Gene therapy caught in more entanglements. Science. Apr 3 1981;212(4490):24-5. doi:10.1126/science.6259731
- 5. Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science. Oct 20 1995;270(5235):475-80. doi:10.1126/science.270.5235.475
- 6. Smith CIE, Blomberg P. [Gene therapy - from idea to reality]. Lakartidningen. Dec 19 2017;114Genterapi - fran ide till verklighet - Annu har fa patienter behandlats och preparaten ar ofta mycket dyra - men utvecklingen gar fort nu.
- 7. Smith KR. Gene Therapy: The Potential Applicability of Gene Transfer Technology to the Human Germline. Int J Med Sci. 2004;1(2):76-91. doi:10.7150/ijms.1.76
- 8. https://pubmed.ncbi.nlm.nih.gov/ 9. Arabi F, Mansouri V, Ahmadbeigi N. Gene therapy clinical trials, where do we go? An overview. Biomed Pharmacother. Sep 2022;153:113324. doi:10.1016/j.biopha.2022.113324
- 10. https://clinicaltrials.gov/.
- 11. Ogbonmide T, Rathore R, Rangrej SB, et al. Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). Cureus. Mar 2023;15(3):e36197. doi:10.7759/cureus.36197
- 12. Eminoglu FT, Koc Yekeduz M, Dogulu N, et al. Inherited metabolic disorders in the neonatal intensive care unit: Red flags to look out for. Pediatr Int. Jan 2022;64(1):e14953. doi:10.1111/ped.14953
- 13. Sugarman EA, Nagan N, Zhu H, et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. Jan 2012;20(1):27-32. doi:10.1038/ejhg.2011.134
- 14. T.R. Ministry of Health 2021a. Premarital Spinal Muscular Atrophy (SMA) Carrier Screening Program. T.R. Ministry of Health. General Directorate of Public Health
- 15. T.R. Ministry of Health 2022. Newborn Metabolic and Endocrine Disease Screening Program. T.R. Ministry of Health. General Directorate of Public Health.
- 16. Waldrop MA, Kolb SJ. Current Treatment Options in Neurology-SMA Therapeutics. Curr Treat Options Neurol. Apr 29 2019;21(6):25. doi:10.1007/s11940-019-0568-z
- 17. Schuster DJ, Dykstra JA, Riedl MS, et al. Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Front Neuroanat. 2014;8:42. doi:10.3389/fnana.2014.00042
- 18. Rashnonejad A, Durmaz B, Özkınay F. The principles of gene therapy and recent advances. Ege Journal of Medicine 2014;53(4):231-240.
Ayrıntılar
| Birincil Dil | İngilizce |
|---|---|
| Konular | Tıbbi Genetik (Kanser Genetiği hariç) |
| Bölüm | Orjinal Araştırma |
| Yazarlar | |
| Yayımlanma Tarihi | 28 Mart 2024 |
| Gönderilme Tarihi | 3 Mart 2024 |
| Kabul Tarihi | 22 Mart 2024 |
| Yayımlandığı Sayı | Yıl 2024 Cilt: 14 Sayı: 2 |
