Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options

@inproceedings{Tanna2017StargardtDC,
  title={Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options},
  author={Preena Tanna and Rupert Wolfgang Strauss and Kaoru Fujinami and Michel Michaelides},
  booktitle={The British journal of ophthalmology},
  year={2017}
}
Stargardt disease (STGD1; MIM 248200) is the most prevalent inherited macular dystrophy and is associated with disease-causing sequence variants in the gene ABCA4 Significant advances have been made over the last 10 years in our understanding of both the clinical and molecular features of STGD1, and also the underlying pathophysiology, which has culminated in ongoing and planned human clinical trials of novel therapies. The aims of this review are to describe the detailed phenotypic and… CONTINUE READING
Recent Discussions
This paper has been referenced on Twitter 11 times over the past 90 days. VIEW TWEETS

From This Paper

Figures, tables, results, connections, and topics extracted from this paper.
21 Extracted Citations
80 Extracted References
Similar Papers

Citing Papers

Publications influenced by this paper.
Showing 1-10 of 21 extracted citations

Referenced Papers

Publications referenced by this paper.
Showing 1-10 of 80 references

Early findings in a Phase I/IIa clinical program for Stargardt disease (STGD1, MIM #248200) [abstract

  • I Audo, R Weleber, T Stout
  • Invest Ophthalmol Vis Sci 2015;56:3819
  • 2015

Similar Papers

Loading similar papers…