Genetic therapeutic strategies for Bardet-Biedl Syndrome

MR Cring - 2020 - search.proquest.com
Abstract Bardet-Beidl Syndrome (BBS) is a pleiotropic ciliopathy that causes a variety of
features in humans and animal models, including retinal degeneration, obesity, male …

Ectopic expression of BBS1 rescues male infertility, but not retinal degeneration, in a BBS1 mouse model

MR Cring, KJ Meyer, CC Searby, A Hedberg-Buenz… - Gene therapy, 2022 - nature.com
Bardet–Biedl syndrome (BBS) is a rare ciliopathy for which there are no current effective
treatments. BBS is a genetically heterogeneous disease, though the M390R mutation in …

Retrotransposon insertion as a novel mutational event in Bardet‐Biedl syndrome

E Tavares, CY Tang, A Vig, S Li… - Molecular genetics & …, 2019 - Wiley Online Library
Abstract Background Bardet‐Biedl syndrome (BBS) is an autosomal recessive pleiotropic
disorder of the primary cilia that leads to severe visual loss in the teenage years …

The Bardet-Biedl Syndrome

VC Sheffield, Q Zhang, E Heon, AV Drack… - Epstein's Inborn Errors …, 2016 - iro.uiowa.edu
Abstract Bardet–Biedl syndrome (BBS)(OMIM 209900) is a heterogeneous autosomal
recessive disorder characterized by clinical findings that include obesity, photoreceptor …

[HTML][HTML] Bardet–biedl syndrome

E Forsythe, PL Beales - European journal of human genetics, 2013 - nature.com
Bardet–Biedl syndrome (BBS) is a rare autosomal recessive ciliopathy characterised by
retinal dystrophy, obesity, post-axial polydactyly, renal dysfunction, learning difficulties and …

Establishing a connection between cilia and Bardet–Biedl syndrome

K Mykytyn, VC Sheffield - Trends in molecular medicine, 2004 - cell.com
Abstract Bardet–Biedl Syndrome (BBS) is a genetic disorder with the primary features of
retinal dystrophy, obesity, polydactyly, structural and functional renal abnormalities, and …

Bardet-Biedl Syndrome: A Comprehensive Review of an Autosomal Recessive Ciliopathy

FV Martin, HMV Jimenez, MEU Reyes… - International Journal of …, 2023 - ijmscr.org
Abstract Bardet-Biedl Syndrome (BBS) is a rare, genetically heterogeneous, and autosomal
recessive ciliopathy characterized by a complex clinical phenotype. This article provides a …

Identification of 28 novel mutations in the Bardet–Biedl syndrome genes: the burden of private mutations in an extensively heterogeneous disease

J Muller, C Stoetzel, MC Vincent, CC Leitch, V Laurier… - Human genetics, 2010 - Springer
Abstract Bardet–Biedl syndrome (BBS), an emblematic disease in the rapidly evolving field
of ciliopathies, is characterized by pleiotropic clinical features and extensive genetic …

A novel splice site variant of the BBS2 gene in a patient with Bardet-Biedl syndrome

H Azizi, M Bonyadi, A Rafat - Human Genome Variation, 2024 - nature.com
Bardet-Biedl syndrome (BBS) is a genetically heterogeneous ciliopathy with autosomal
recessive inheritance and an estimated worldwide prevalence of 1 in 150,000 individuals …

[HTML][HTML] Mutation profile of BBS genes in Iranian patients with Bardet–Biedl syndrome: genetic characterization and report of nine novel mutations in five BBS genes

Z Fattahi, P Rostami, A Najmabadi, M Mohseni… - Journal of human …, 2014 - nature.com
Bardet–Biedl syndrome (BBS) is a rare ciliopathy disorder that is clinically and genetically
heterogeneous with 18 known genes. This study was performed to characterize responsible …