An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.

Autor: Keller CG; Novartis Institutes for Biomedical Research, Basel, Switzerland., Shin Y; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Monteys AM; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA., Renaud N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Beibel M; Novartis Institutes for Biomedical Research, Basel, Switzerland., Teider N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Peters T; Novartis Institutes for Biomedical Research, Basel, Switzerland., Faller T; Novartis Institutes for Biomedical Research, Basel, Switzerland., St-Cyr S; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Knehr J; Novartis Institutes for Biomedical Research, Basel, Switzerland., Roma G; Novartis Institutes for Biomedical Research, Basel, Switzerland., Reyes A; Novartis Institutes for Biomedical Research, Basel, Switzerland., Hild M; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Lukashev D; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Theil D; Novartis Institutes for Biomedical Research, Basel, Switzerland., Dales N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Cha JH; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Borowsky B; Novartis Pharmaceuticals, East Hanover, NJ, USA., Dolmetsch R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Davidson BL; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu., Sivasankaran R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA. rajeev.sivasankaran@novartis.com.

Publikováno v: Nature communications [Nat Commun] 2022 Mar 03; Vol. 13 (1), pp. 1150. Date of Electronic Publication: 2022 Mar 03.

Regulated control of gene therapies by drug-induced splicing.

Autor: Monteys AM; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. monteysam@chop.edu.; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA. monteysam@chop.edu., Hundley AA; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Ranum PT; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Tecedor L; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Muehlmatt A; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Lim E; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Lukashev D; Neuroscience Disease Area, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, USA., Sivasankaran R; Neuroscience Disease Area, Novartis Institutes for BioMedical Research (NIBR), Cambridge, MA, USA., Davidson BL; Raymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu.

Publikováno v: Nature [Nature] 2021 Aug; Vol. 596 (7871), pp. 291-295. Date of Electronic Publication: 2021 Jul 28.

CAR T Cell Nanosymbionts: Revealing the Boundless Potential of a New Dyad.

Autor: Baena, Juan C.^1,2 (AUTHOR) juan.baena@fvl.org.com, Pérez, Lucy M.^1,2 (AUTHOR) alejandro.toro1@u.icesi.edu.com, Toro-Pedroza, Alejandro^1,2 (AUTHOR), Kitawaki, Toshio³ (AUTHOR) kitawaki@kuhp.kyoto-u.ac.jp, Loukanov, Alexandre^4,5 (AUTHOR) loukanov@gunma.kosen-ac.jp

Publikováno v: International Journal of Molecular Sciences. Dec2024, Vol. 25 Issue 23, p13157. 31p.

Changes in immune cell populations during acclimatization to high altitude.

Autor: Pham, Kathy¹ (AUTHOR), Vargas, Abel¹ (AUTHOR), Frost, Shyleen¹ (AUTHOR), Shah, Saheli¹ (AUTHOR), Heinrich, Erica C.¹ (AUTHOR) erica.heinrich@medsch.ucr.edu

Publikováno v: Physiological Reports. Nov2024, Vol. 12 Issue 22, p1-15. 15p.

Caffeine: A Multifunctional Efficacious Molecule with Diverse Health Implications and Emerging Delivery Systems.

Autor: Song, Xinjie¹ (AUTHOR) foodtech.song@foxmail.com, Singh, Mahendra² (AUTHOR) m.singh2685@gmail.com, Lee, Kyung Eun³ (AUTHOR) keun126@ynu.ac.kr, Vinayagam, Ramachandran² (AUTHOR) rambio@ynu.ac.kr, Kang, Sang Gu² (AUTHOR) rambio@ynu.ac.kr

Publikováno v: International Journal of Molecular Sciences. Nov2024, Vol. 25 Issue 22, p12003. 32p.