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MDA5 deficiency

MedGen UID:
1642875
Concept ID:
C4708512
Disease or Syndrome
Synonyms: IFIH1 (interferon induced with helicase C domain 1) deficiency; MDA5 (melanoma differentiation-associated gene 5) deficiency; Melanoma differentiation-associated gene 5 deficiency
SNOMED CT: MDA5 (melanoma differentiation-associated gene 5) deficiency (768560008); IFIH1 (interferon induced with helicase C domain 1) deficiency (768560008); Melanoma differentiation-associated gene 5 deficiency (768560008); MDA5 deficiency (768560008)

Definition

An immunodeficiency disorder with onset in infancy that leads to recurrent, severe infections of the respiratory tract. Infections are most frequently caused by rhinovirus. Respiratory syncytial virus and the influenza virus may also cause recurrent infections. Infection may require hospital admission and repeated infection can lead to chronic lung disease. Infections usually become less frequent with maturity. Caused by mutations in the IFIH1 gene, which provides instructions for making the MDA5 protein. Deficiency of MDA5 protein activity reduces interferon production in response to RNA-containing viruses. The inheritance pattern is unclear. In some cases, the condition seems to follow an autosomal recessive pattern, in other cases it appears that the condition is inherited in an autosomal dominant pattern. [from SNOMEDCT_US]

Professional guidelines

PubMed

Gupta L, Muhammed H, Naveen R, Kharbanda R, Gangadharan H, Misra DP, Lilleker JB, Chinoy H, Agarwal V
Rheumatol Int 2020 Dec;40(12):2047-2055. Epub 2020 Sep 2 doi: 10.1007/s00296-020-04695-1. PMID: 32880031

Recent clinical studies

Diagnosis

Cananzi M, Wohler E, Marzollo A, Colavito D, You J, Jing H, Bresolin S, Gaio P, Martin R, Mescoli C, Bade S, Posey JE, Dalle Carbonare M, Tung W, Jhangiani SN, Bosa L, Zhang Y, Filho JS, Gabelli M, Kellermayer R, Kader HA, Oliva-Hemker M, Perilongo G, Lupski JR, Biffi A, Valle D, Leon A, de Macena Sobreira NL, Su HC, Guerrerio AL
Hum Genet 2021 Sep;140(9):1299-1312. Epub 2021 Jun 29 doi: 10.1007/s00439-021-02300-4. PMID: 34185153Free PMC Article

Therapy

Cananzi M, Wohler E, Marzollo A, Colavito D, You J, Jing H, Bresolin S, Gaio P, Martin R, Mescoli C, Bade S, Posey JE, Dalle Carbonare M, Tung W, Jhangiani SN, Bosa L, Zhang Y, Filho JS, Gabelli M, Kellermayer R, Kader HA, Oliva-Hemker M, Perilongo G, Lupski JR, Biffi A, Valle D, Leon A, de Macena Sobreira NL, Su HC, Guerrerio AL
Hum Genet 2021 Sep;140(9):1299-1312. Epub 2021 Jun 29 doi: 10.1007/s00439-021-02300-4. PMID: 34185153Free PMC Article
Jing H, Su HC
Curr Opin Pediatr 2019 Dec;31(6):815-820. doi: 10.1097/MOP.0000000000000827. PMID: 31693592Free PMC Article

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