Total submissions: 4
Submitter | RCV | SCV | Clinical significance | Condition | Last evaluated | Review status | Method | Comment |
---|---|---|---|---|---|---|---|---|
CFTR2 | RCV000577043 | SCV001981593 | pathogenic | Cystic fibrosis | 2020-01-10 | reviewed by expert panel | research | |
CFTR- |
RCV001009373 | SCV001169226 | pathogenic | Cystic fibrosis; CFTR-related disorder | 2018-01-29 | criteria provided, single submitter | curation | when the variant is in trans with another CF-causing variation, can either result in CF or in a CFTR-RD |
Women's Health and Genetics/Laboratory Corporation of America, |
RCV000577043 | SCV001737621 | likely pathogenic | Cystic fibrosis | 2021-05-20 | criteria provided, single submitter | clinical testing | Variant summary: CFTR c.1801A>T (p.Ile601Phe) results in a non-conservative amino acid change located in the ABC transporter-like of the encoded protein sequence. Five of five in-silico tools predict a damaging effect of the variant on protein function. The variant allele was found at a frequency of 4.3e-06 in 233316 control chromosomes. c.1801A>T has been reported in the literature in individuals affected with CFTR-Related Diseases (CFTR-RD), including at least one patient with pancreatic sufficient CF and one patient with CBAVD (Vankeerberghen_1998, Steiner_2011). The variant has also been reported in 5 patients in the CFTR2 database, and was reported in the CFTR-France database in 2 patients with CF and 4 patients with CFTR-RD. At least one functional study reported that the variant gave rise to a protein that was aberrantly processed, resulting in the absence of sufficient CFTR protein at the cell surface (Vankeerberghen_1998). The CFTR2 and CFTR-France databases both report the variant as pathogenic for CF and/or CFTR-RD. However, the working group of the French CF society cited the variant in recently published guidelines as "a mutation of unproved or uncertain clinical relevance" (Sermet-Gaudelus_2017). Based on the evidence outlined above, the variant was classified as likely pathogenic. |
Clin |
RCV000577043 | SCV000678964 | not provided | Cystic fibrosis | no assertion provided | literature only |