Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine model.

Autor: Zhao XJ; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Mohsen AW; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, 15261, USA., Mihalik S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Solo K; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Basu S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Aliu E; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Shi H; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Kochersberger C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Karunanidhi A; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Van't Land C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA., Coughlan KA; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Siddiqui S; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Rice LM; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Hillier S; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Guadagnin E; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., DeAntonis C; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Giangrande PH; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Martini PGV; Moderna Therapeutics, Rare Diseases, Cambridge, MA, 02139, USA., Vockley J; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, 15224, USA.; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, 15261, USA.

Publikováno v: Human molecular genetics [Hum Mol Genet] 2023 Jul 04; Vol. 32 (14), pp. 2347-2356.

Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model.

Autor: Zhao XJ; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Mohsen AW; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, USA., Mihalik S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Solo K; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Aliu E; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Shi H; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Basu S; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Kochersperger C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Van't Land C; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Karunanidhi A; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA., Coughlan KA; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Siddiqui S; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Rice LM; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Hillier S; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Guadagnin E; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Giangrande PH; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Martini PGV; Moderna Therapeutics, Inc., Rare Diseases, 200 Technology Square, Cambridge, MA, USA., Vockley J; Division of Genetic and Genomic Medicine, Department of Pediatrics, University of Pittsburgh, Pittsburgh, PA, USA; Department of Human Genetics, University of Pittsburgh, Pittsburgh, PA, USA. Electronic address: vockleyg@upmc.edu.

Publikováno v: Molecular genetics and metabolism [Mol Genet Metab] 2023 Jan; Vol. 138 (1), pp. 106982. Date of Electronic Publication: 2022 Dec 23.

Neuroinflammatory Proteins in Huntington's Disease: Insights into Mechanisms, Diagnosis, and Therapeutic Implications.

Autor: Li, Xinhui¹ (AUTHOR) lxh1996@stu2022.jnu.edu.cn, Tong, Huichun^1,2 (AUTHOR) tonghuichun@stu2021.jnu.edu.cn, Xu, Shuying¹ (AUTHOR) xushuying@stu.jnu.edu.cn, Zhou, Gongke¹ (AUTHOR) zgk00@stu2022.jnu.edu.cn, Yang, Tianqi¹ (AUTHOR) yangtianqi12138@stu2021.jnu.edu.cn, Yin, Shurui¹ (AUTHOR) yinshurui20040416@stu2022.jnu.edu.cn, Yang, Sitong¹ (AUTHOR) yangsitong@stu2023.jnu.edu.cn, Li, Xiaojiang¹ (AUTHOR) xjli33@jnu.edu.cn, Li, Shihua¹ (AUTHOR) lishihualis@jnu.edu.cn

Publikováno v: International Journal of Molecular Sciences. Nov2024, Vol. 25 Issue 21, p11787. 26p.

Geographic monitoring for early disease detection (GeoMEDD).

Autor: Curtis A; GIS Health & Hazards Lab, Department of Population and Quantitative Health Sciences, School of Medicine, Case Western Reserve University, Cleveland, OH, USA., Ajayakumar J; GIS Health & Hazards Lab, Department of Population and Quantitative Health Sciences, School of Medicine, Case Western Reserve University, Cleveland, OH, USA., Curtis J; GIS Health & Hazards Lab, Department of Population and Quantitative Health Sciences, School of Medicine, Case Western Reserve University, Cleveland, OH, USA. jacqueline.curtis@case.edu., Mihalik S; University Hospitals Health System, Cleveland, OH, USA., Purohit M; University Hospitals Health System, Cleveland, OH, USA., Scott Z; University Hospitals Health System, Cleveland, OH, USA., Muisyo J; University Hospitals Health System, Cleveland, OH, USA., Labadorf J; University Hospitals Health System, Cleveland, OH, USA., Vijitakula S; University Hospitals Health System, Cleveland, OH, USA., Yax J; University Hospitals Health System, Cleveland, OH, USA., Goldberg DW; GeoInnovation Service Center, Department of Geography, Texas A&M University, College Station, TX, USA.

Publikováno v: Scientific reports [Sci Rep] 2020 Dec 10; Vol. 10 (1), pp. 21753. Date of Electronic Publication: 2020 Dec 10.

Metabolic Reprogramming of Astrocytes in Pathological Conditions: Implications for Neurodegenerative Diseases.

Autor: Calì, Corrado^1,2 (AUTHOR) corrado.cali@unito.it, Cantando, Iva³ (AUTHOR) iva.cantando@unil.ch, Veloz Castillo, Maria Fernanda^1,2,4 (AUTHOR), Gonzalez, Laurine³ (AUTHOR) laurine.gonzalez@unil.ch, Bezzi, Paola^3,5 (AUTHOR) corrado.cali@unito.it

Publikováno v: International Journal of Molecular Sciences. Aug2024, Vol. 25 Issue 16, p8922. 22p.