Literature DB >> 25984432

Can Hutchinson-Gilford progeria syndrome be cured in the future?

Neeha Abdul Rehman1, Aneeqa Abdul Rehman1, Isra Najib Ashraf1, Shahrukh Ahmed1.   

Abstract

Progeria is a rare genetic disease that manifests with progressive symptoms eventually leading to death. The only current treatment protocol of such patients is symptom based. However, recent trials are testing potential and promising drugs to treat the underlying genetic mutation and increase life expectancy of such patients.

Entities:  

Keywords:  Cure; Progeria; farnesyltransferase inhibitors

Year:  2015        PMID: 25984432      PMCID: PMC4428187          DOI: 10.5582/irdr.2015.01003

Source DB:  PubMed          Journal:  Intractable Rare Dis Res        ISSN: 2186-3644


  4 in total

1.  Rapamycin reverses cellular phenotypes and enhances mutant protein clearance in Hutchinson-Gilford progeria syndrome cells.

Authors:  Kan Cao; John J Graziotto; Cecilia D Blair; Joseph R Mazzulli; Michael R Erdos; Dimitri Krainc; Francis S Collins
Journal:  Sci Transl Med       Date:  2011-06-29       Impact factor: 17.956

2.  Blocking protein farnesylation improves nuclear shape abnormalities in keratinocytes of mice expressing the prelamin A variant in Hutchinson-Gilford progeria syndrome.

Authors:  Yuexia Wang; Cecilia Ostlund; Howard J Worman
Journal:  Nucleus       Date:  2010 Sep-Oct       Impact factor: 4.197

3.  Targeting isoprenylcysteine methylation ameliorates disease in a mouse model of progeria.

Authors:  Mohamed X Ibrahim; Volkan I Sayin; Murali K Akula; Meng Liu; Loren G Fong; Stephen G Young; Martin O Bergo
Journal:  Science       Date:  2013-05-16       Impact factor: 47.728

4.  Neurologic features of Hutchinson-Gilford progeria syndrome after lonafarnib treatment.

Authors:  Nicole J Ullrich; Mark W Kieran; David T Miller; Leslie B Gordon; Yoon-Jae Cho; V Michelle Silvera; Anita Giobbie-Hurder; Donna Neuberg; Monica E Kleinman
Journal:  Neurology       Date:  2013-06-28       Impact factor: 9.910

  4 in total

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