![](/images/graphics-bg.png)
Oligonucleotide-Based Therapy for FTDALS Caused by the C9orf72 Repeat Expansion : A Perspective
المؤلفون المشاركون
Varela, Miguel A.
Wood, Matthew J. A.
Douglas, Andrew G. L.
Fernandes, Stephanie A.
Aoki, Yoshitsugu
المصدر
العدد
المجلد 2013، العدد 2013 (31 ديسمبر/كانون الأول 2013)، ص ص. 1-11، 11ص.
الناشر
Hindawi Publishing Corporation
تاريخ النشر
2013-11-17
دولة النشر
مصر
عدد الصفحات
11
التخصصات الرئيسية
الملخص EN
Amyotrophic lateral sclerosis (ALS) is a progressive and lethal disease of motor neuron degeneration, leading to paralysis of voluntary muscles and death by respiratory failure within five years of onset.
Frontotemporal dementia (FTD) is characterised by degeneration of frontal and temporal lobes, leading to changes in personality, behaviour, and language, culminating in death within 5–10 years.
Both of these diseases form a clinical, pathological, and genetic continuum of diseases, and this link has become clearer recently with the discovery of a hexanucleotide repeat expansion in the C9orf72 gene that causes the FTD/ALS spectrum, that is, c9FTD/ALS.
Two basic mechanisms have been proposed as being potentially responsible for c9FTD/ALS: loss-of-function of the protein encoded by this gene (associated with aberrant DNA methylation) and gain of function through the formation of RNA foci or protein aggregates.
These diseases currently lack any cure or effective treatment.
Antisense oligonucleotides (ASOs) are modified nucleic acids that are able to silence targeted mRNAs or perform splice modulation, and the fact that they have proved efficient in repeat expansion diseases including myotonic dystrophy type 1 makes them ideal candidates for c9FTD/ALS therapy.
Here, we discuss potential mechanisms and challenges for developing oligonucleotide-based therapy for c9FTD/ALS.
نمط استشهاد جمعية علماء النفس الأمريكية (APA)
Fernandes, Stephanie A.& Douglas, Andrew G. L.& Varela, Miguel A.& Wood, Matthew J. A.& Aoki, Yoshitsugu. 2013. Oligonucleotide-Based Therapy for FTDALS Caused by the C9orf72 Repeat Expansion : A Perspective. Journal of Nucleic Acids،Vol. 2013, no. 2013, pp.1-11.
https://search.emarefa.net/detail/BIM-454633
نمط استشهاد الجمعية الأمريكية للغات الحديثة (MLA)
Fernandes, Stephanie A.…[et al.]. Oligonucleotide-Based Therapy for FTDALS Caused by the C9orf72 Repeat Expansion : A Perspective. Journal of Nucleic Acids No. 2013 (2013), pp.1-11.
https://search.emarefa.net/detail/BIM-454633
نمط استشهاد الجمعية الطبية الأمريكية (AMA)
Fernandes, Stephanie A.& Douglas, Andrew G. L.& Varela, Miguel A.& Wood, Matthew J. A.& Aoki, Yoshitsugu. Oligonucleotide-Based Therapy for FTDALS Caused by the C9orf72 Repeat Expansion : A Perspective. Journal of Nucleic Acids. 2013. Vol. 2013, no. 2013, pp.1-11.
https://search.emarefa.net/detail/BIM-454633
نوع البيانات
مقالات
لغة النص
الإنجليزية
الملاحظات
Includes bibliographical references
رقم السجل
BIM-454633
قاعدة معامل التأثير والاستشهادات المرجعية العربي "ارسيف Arcif"
أضخم قاعدة بيانات عربية للاستشهادات المرجعية للمجلات العلمية المحكمة الصادرة في العالم العربي
![](/images/ebook-kashef.png)
تقوم هذه الخدمة بالتحقق من التشابه أو الانتحال في الأبحاث والمقالات العلمية والأطروحات الجامعية والكتب والأبحاث باللغة العربية، وتحديد درجة التشابه أو أصالة الأعمال البحثية وحماية ملكيتها الفكرية. تعرف اكثر
![](/images/kashef-image.png)