Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation | Zendy

Céline J. Rocca | Zendy; Alexander Kreymerman | Zendy; Sarah N. Ur | Zendy; Katie Frizzi | Zendy; Swati Naphade | Zendy; Athena Lau | Zendy; Tammy Tran | Zendy; Nigel A. Calcutt | Zendy; Jeffrey L. Goldberg | Zendy; Stéphanie Cherqui | Zendy

AI Assistant Blog Pricing

Home ZAIA Blog

Open Access

Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation

Author(s) -

Céline J. Rocca,

Alexander Kreymerman,

Sarah N. Ur,

Katie Frizzi,

Swati Naphade,

Athena Lau,

Tammy Tran,

Nigel A. Calcutt,

Jeffrey L. Goldberg,

Stéphanie Cherqui

Publication year - 2015

Publication title -

investigative ophthalmology and visual science

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.935

H-Index - 218

eISSN - 1552-5783

pISSN - 0146-0404

DOI - 10.1167/iovs.15-17107

Subject(s) - cystinosis , transplantation , progenitor cell , cornea , stem cell , biology , corneal transplantation , haematopoiesis , microbiology and biotechnology , cancer research , pathology , immunology , medicine , cystine , biochemistry , cysteine , neuroscience , enzyme

Cystinosis is caused by a deficiency in the lysosomal cystine transporter, cystinosin (CTNS gene), resulting in cystine crystal accumulation in tissues. In eyes, crystals accumulate in the cornea causing photophobia and eventually blindness. Hematopoietic stem progenitor cells (HSPCs) rescue the kidney in a mouse model of cystinosis. We investigated the potential for HSPC transplantation to treat corneal defects in cystinosis.

The content you want is available to Zendy users.

Already have an account? Click here to sign in.

Having issues? You can contact us here

Accelerating Research