عنوان

Models of TDP-43 Proteinopathy in ALS / FTD and RNA targeting using CRISPR / Cas9

شماره

TL35k1q39z

زبان متن نوشتاري يا گفتاري و مانند آن

انگلیسی

عنوان اصلي

Models of TDP-43 Proteinopathy in ALS / FTD and RNA targeting using CRISPR / Cas9

نام عام مواد

[Thesis]

نام نخستين پديدآور

Pirie, Elaine Christine

نام ساير پديدآوران

Ravits, John MYeo, Eugene W

تاریخ نشرو بخش و غیره

2017

کسي که مدرک را اعطا کرده

Ravits, John MYeo, Eugene W

امتياز متن

2017

متن يادداشت

Misregulation of RNA processing is a major component of the related neurodegenerative diseases Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. TDP-43 is an RNA binding protein and the primary pathological finding in both diseases. Post-translational modification of TDP-43 by direct S-nitrosylation of cysteines 173 and 175 leads to disulfide bond formation and loss of solubility in ALS / FTD models. Further, TDP-43 aggregation may be able to induce nitrosative stress within the cell, leading to further loss of solubility. We present models of TDP-43 aggregation that demonstrate TDP-43 is capable of propagating between cells quantifiably, spreading disease-associated aggregates. C9orf72 is the most common genetic cause of ALS / FTD, and here we establish a CRISPR / Cas9 technology capable of tracking and degrading RNA foci found in in myotonic dystrophy and C9 ALS. Our findings provide a novel framework for therapeutics targeted at TDP-43 aggregation and RNA toxicity.

مستند نام اشخاص تاييد نشده

Shrodes, Addie C.

مستند نام اشخاص تاييد نشده

Pirie, Elaine Christine

مستند نام تنالگان تاييد نشده

UC San Diego

نام الکترونيکي

فرمت انتشار

p

نوع ماده

[Thesis]

کد کاربرگه

276903

سطح دسترسي

a

تكميل شده

Y