How close are we to therapies for Sanfilippo disease?

Lidia Gaffke , Karolina Pierzynowska , Ewa Piotrowska , Grzegorz Węgrzyn

Abstract

n/a
Author Lidia Gaffke (FB / DMoB)
Lidia Gaffke,,
- Department of Molecular Biology
, Karolina Pierzynowska (FB / DMoB)
Karolina Pierzynowska,,
- Department of Molecular Biology
, Ewa Piotrowska (FB / DMoB)
Ewa Piotrowska,,
- Department of Molecular Biology
, Grzegorz Węgrzyn (FB / DMoB)
Grzegorz Węgrzyn,,
- Department of Molecular Biology
Journal seriesMetabolic Brain Disease, ISSN 0885-7490, (A 20 pkt)
Issue year2018
Vol33
No1
Pages1-10
Publication size in sheets0.5
Keywords in EnglishSanfilippo disease, neurodegeneration, lysosomal storage, enzyme replacement therapy, gene therapy, substrate reduction therapy
DOIDOI:10.1007/s11011-017-0111-4
URL https://link.springer.com/content/pdf/10.1007%2Fs11011-017-0111-4.pdf
Languageen angielski
LicenseOther; published final; Uznanie Autorstwa (CC-BY); with publication
Score (nominal)25
ScoreMinisterial score = 20.0, ArticleFromJournal
Ministerial score (2013-2016) = 25.0, ArticleFromJournal
Publication indicators Scopus Citations = 2; WoS Citations = 2; WoS Impact Factor: 2017 = 2.441 (2) - 2017=2.481 (5)
Citation count*4 (2019-04-22)
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* presented citation count is obtained through Internet information analysis and it is close to the number calculated by the Publish or Perish system.
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