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Treacher Collins syndrome (TCS) is a frequent cause of mandibulofacial dysostosis. To date, TCS-causing mutations in three genes, namely TCOF1, POLR1D, and POLR1C have been identified. TCS is usually inherited in an autosomal dominant manner, with a high clinical variability and no phenotype-genotype correlation. Up-to now, five families have been reported with an autosomal recessive mode of inheritance due to mutations in POLR1D or POLR1C. We report here a new family with two sisters affected by mild TCS carrying compound POLR1C heterozygous mutations, and review the literature on mild forms of TCS, autosomal recessive inheritance in this syndrome and POLR1C mutations.
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http://dx.doi.org/10.1002/ajmg.a.61147 | DOI Listing |
PLoS One
September 2025
HUN-REN Centre for Ecological Research, Institute of Evolution, Budapest, Hungary.
We develop a model that integrates evolutionary matrix game theory with Mendelian genetics. Within this framework, we define the genotype dynamics that describes how the frequencies of genotypes change in sexual diploid populations. We show that our formal definition of evolutionary stability for genotype distributions implies the stability of the corresponding interior equilibrium point in the genotype dynamics.
View Article and Find Full Text PDFJ Pediatr Hematol Oncol
September 2025
Children's Hospital of Michigan, Division of Hematology/Oncology.
Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet disorder characterized by abnormalities in platelet aggregation, resulting from quantitative or qualitative defects in integrins αIIb and β3. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only potentially curative therapeutic approach for severe GT. In this report, we present 2 children with GT that underwent successful allo-HSCT, along with 2008 to 2022 data from the Center for International Blood and Marrow Transplant Research and a summary of the existing literature providing further evidence that allo-HSCT can be a curative approach that prevents severe and life-threatening bleeding in GT.
View Article and Find Full Text PDFMol Ther Methods Clin Dev
June 2025
Shanghai Vitalgen BioPharma Co., Ltd., Shanghai 201210, China.
Bietti crystalline dystrophy (BCD) is an autosomal recessive disorder caused by loss-of-function mutations in the gene, characterized by crystal-like lipid deposits in the retina, progressive photoreceptor loss, and retinal pigment epithelium (RPE) deterioration. Currently, there are no approved treatments for BCD. VGR-R01, an investigational gene therapy, uses subretinal administration of recombinant adeno-associated virus type 8 (AAV8) vector to deliver the human CYP4V2 gene.
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