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choroideremia

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Also known as progressive Choroidal Atrophy, CHM

Choroideremia (; CHM) is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life. Progression continues throughout the individual's life, but both the rate of change and the degree of visual loss are variable among those affected, even within the same family.

Key facts

Medical condition (new).name
Choroideremia
Medical condition (new).synonyms
CHM, Tapetochoroidal dystrophy
Medical condition (new).image
File:Sex linked inheritance.png
Medical condition (new).caption
An example pedigree chart, showing the inheritance of a sex-linked disorder like choroideremia.
Medical condition (new).field
Ophthalmology
Medical condition (new).symptoms
Progressive vision loss
Medical condition (new).onset
Childhood
Medical condition (new).duration
Chronic
Medical condition (new).causes
Genetic
Medical condition (new).diagnosis
Genetic testing, family history, fundus examination

via Wikipedia infobox

Research

782 papers

via PubMed

~10 min read

Encyclopedic overview

14 sections
Contents
  • Presentation
  • Cause
  • Diagnosis
  • Management
  • Gene therapy
  • Preimplantation genetic diagnosis
  • Other potential therapies
  • Research
  • History
  • Basic research
  • Culture
  • References
  • Further reading
  • External links

Choroideremia (; CHM) is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life. Progression continues throughout the individual's life, but both the rate of change and the degree of visual loss are variable among those affected, even within the same family.

Choroideremia is caused by a loss-of-function mutation in the CHM gene which encodes Rab escort protein 1 (REP1), a protein involved in lipid modification of Rab proteins. While the complete mechanism of disease is not fully understood, the lack of a functional protein in the retina results in cell death and the gradual deterioration of the retinal pigment epithelium (RPE), photoreceptors and the choroid.

Excerpted from Wikipedia’s “choroideremia” article, available under the CC BY-SA 4.0 licence.