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A targeted gene panel illuminates pathogenesis in young people with unexplained kidney failure.
Beal F, Forrester N, Watson E, Williams M, Buckton A, Marlais M, Maxted A; UK Gene Panel Study Group; Woolf AS, Saleem MA, Platt C. Beal F, et al. J Nephrol. 2024 Jun;37(5):1273-1284. doi: 10.1007/s40620-024-01964-1. Epub 2024 Jun 5. J Nephrol. 2024. PMID: 38837003
Positive predictors of a genetic diagnosis included a family history of renal disease (60% of positive cases) and extra-renal disease manifestations (48% of positive cases). CONCLUSION: This is the first study to evaluate the R257 gene panel in unexplained young-onset kidn …
Positive predictors of a genetic diagnosis included a family history of renal disease (60% of positive cases) and extra-renal disease …
Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.
Griesenbach U, Alton EW; UK Cystic Fibrosis Gene Therapy Consortium. Griesenbach U, et al. Adv Drug Deliv Rev. 2009 Feb 27;61(2):128-39. doi: 10.1016/j.addr.2008.09.010. Epub 2008 Dec 24. Adv Drug Deliv Rev. 2009. PMID: 19138713 Review.
Gene therapy is currently being developed for a wide range of acute and chronic lung diseases. The target cells, and to a degree the extra and intra-cellular barriers, are disease-specific and over the past decade the gene therapy community has recognized that no one vecto …
Gene therapy is currently being developed for a wide range of acute and chronic lung diseases. The target cells, and to a degree the extr