G-Quadruplex RNA Based PROTAC Enables Targeted Degradation of RNA Binding Protein FMRP for Tumor Immunotherapy.

Autor: Peng R; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Huang Q; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Wang L; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Qiao G; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Huang X; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Jiang J; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China., Chu X; State Key Laboratory of Chemo/Bio-Sensing and Chemometrics, College of Chemistry and Chemical Engineering, Hunan University, 410082, Changsha, P. R. China.

Publikováno v: Angewandte Chemie (International ed. in English) [Angew Chem Int Ed Engl] 2024 Nov 18; Vol. 63 (47), pp. e202402715. Date of Electronic Publication: 2024 Oct 14.

FMRP regulates MFF translation to locally direct mitochondrial fission in neurons.

Autor: Fenton AR; Department of Genetics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Department of Physiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Cell and Molecular Biology Graduate Group, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Pennsylvania Muscle Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Peng R; Department of Biochemistry and Biophysics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Institute of Structural Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Bond C; Department of Physiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Cell and Molecular Biology Graduate Group, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Hugelier S; Department of Physiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Lakadamyali M; Department of Physiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Cell and Molecular Biology Graduate Group, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Pennsylvania Muscle Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Chang YW; Pennsylvania Muscle Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Department of Biochemistry and Biophysics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.; Institute of Structural Biology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA., Holzbaur ELF; Department of Physiology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA. holzbaur@pennmedicine.upenn.edu.; Cell and Molecular Biology Graduate Group, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA. holzbaur@pennmedicine.upenn.edu.; Pennsylvania Muscle Institute, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA. holzbaur@pennmedicine.upenn.edu., Jongens TA; Department of Genetics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA. jongens@upenn.edu.; Cell and Molecular Biology Graduate Group, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA. jongens@upenn.edu.

Publikováno v: Nature cell biology [Nat Cell Biol] 2024 Dec; Vol. 26 (12), pp. 2061-2074. Date of Electronic Publication: 2024 Nov 15.

METTL3-Mediated m ⁶ A Modification of FMRP Drives Hepatocellular Carcinoma Progression and Indicates Poor Prognosis.

Autor: Fu S; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Sun D; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Wang Z; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Zhu P; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Ding W; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Huang J; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Guo X; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Yang Y; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China., Gu F; The Third Department of Hepatic Surgery, Eastern Hepatobiliary Surgery Hospital, Second Military Medical University (Naval Medical University), Shanghai, China.

Publikováno v: Cancer biotherapy & radiopharmaceuticals [Cancer Biother Radiopharm] 2024 Dec; Vol. 39 (10), pp. 745-754. Date of Electronic Publication: 2024 Sep 12.

Ascorbic Acid Ameliorates Molecular and Developmental Defects in Human-Induced Pluripotent Stem Cell and Cerebral Organoid Models of Fragile X Syndrome.

Autor: Gunapala KM; Department of Biomedicine, University of Basel, Mattenstrasse 28, 4058 Basel, Switzerland.; The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Institute of Life Sciences, The Hebrew University, Jerusalem 91904, Israel., Gadban A; The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Institute of Life Sciences, The Hebrew University, Jerusalem 91904, Israel., Noreen F; Department of Biomedicine, University of Basel, Mattenstrasse 28, 4058 Basel, Switzerland.; Swiss Institute of Bioinformatics, 4031 Basel, Switzerland., Schär P; Department of Biomedicine, University of Basel, Mattenstrasse 28, 4058 Basel, Switzerland., Benvenisty N; The Azrieli Center for Stem Cells and Genetic Research, Department of Genetics, Institute of Life Sciences, The Hebrew University, Jerusalem 91904, Israel., Taylor V; Department of Biomedicine, University of Basel, Mattenstrasse 28, 4058 Basel, Switzerland.

Publikováno v: International journal of molecular sciences [Int J Mol Sci] 2024 Nov 26; Vol. 25 (23). Date of Electronic Publication: 2024 Nov 26.

Modeling antisense oligonucleotide therapy in MECP2 duplication syndrome human iPSC-derived neurons reveals gene expression programs responsive to MeCP2 levels.

Autor: Bajikar SS; Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States.; Department of Cell Biology, University of Virginia, 1340 Jefferson Park Avenue, Charlottesville, VA 22903, United States.; Department of Biomedical Engineering, University of Virginia, 415 Lane Road, Charlottesville, VA 22903, United States., Sztainberg Y; Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States., Trostle AJ; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States.; Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States., Tirumala HP; Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States., Wan YW; Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States., Harrop CL; Department of Cell Biology, University of Virginia, 1340 Jefferson Park Avenue, Charlottesville, VA 22903, United States., Bengtsson JD; Pacific Northwest Research Institute, 720 Broadway, Seattle, WA 98122, United States., Carvalho CMB; Pacific Northwest Research Institute, 720 Broadway, Seattle, WA 98122, United States., Pehlivan D; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States.; Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Section of Neurology and Developmental Neuroscience, Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Texas Children's Hospital, 6621 Fannin Street, Houston, TX 77030, United States., Suter B; Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Section of Neurology and Developmental Neuroscience, Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Texas Children's Hospital, 6621 Fannin Street, Houston, TX 77030, United States., Neul JL; Vanderbilt Kennedy Center, 110 Magnolia Circle, Vanderbilt University Medical Center, Nashville, TN 37232, United States., Liu Z; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States.; Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States., Jafar-Nejad P; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, United States., Rigo F; Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, United States., Zoghbi HY; Department of Molecular and Human Genetics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Jan and Dan Duncan Neurological Research Institute at Texas Children's Hospital, 1250 Moursund Street, Houston, TX 77030, United States.; Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Section of Neurology and Developmental Neuroscience, Department of Pediatrics, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, United States.; Texas Children's Hospital, 6621 Fannin Street, Houston, TX 77030, United States.; Howard Hughes Medical Institute, Baylor College of Medicine, Houston, TX 77030, United States.

Publikováno v: Human molecular genetics [Hum Mol Genet] 2024 Nov 08; Vol. 33 (22), pp. 1986-2001.