المؤلفون: Calzoni, Eleonora, Cerrotti, Giada, Sagini, Krizia, Delo, Federica, Buratta, Sandra, Pellegrino, Roberto Maria, Alabed, Husam B. R., Fratini, Federica, Emiliani, Carla, Urbanelli, Lorena

المصدر: Journal of Functional Biomaterials; Jun2024, Vol. 15 Issue 6, p153, 13p

مصطلحات موضوعية: EXTRACELLULAR vesicles, ENZYME replacement therapy, LYSOSOMAL storage diseases, ENZYME deficiency, THERAPEUTICS, POLYMERSOMES

المؤلفون: Jiménez-Bolaños, Rayco, Hernández-Fernández, Francisco, García-García, Jorge, Ayo-Martín, Óscar, del Rey Megias, Laura, Molina-Nuevo, Juan David, Segura, Tomás

المصدر: Journal of Medical Case Reports; 5/12/2024, Vol. 18 Issue 1, p1-4, 4p

مصطلحات موضوعية: THERAPEUTICS, ENDOVASCULAR surgery, ISCHEMIC stroke, LYSOSOMAL storage diseases, HYPERTROPHIC cardiomyopathy, GLYCOGEN storage disease type II

المؤلفون: Hui-An Chen, Rai-Hseng Hsu, Ching-Ya Fang, Desai, Ankit K., Ni-Chung Lee, Wuh-Liang Hwu, Fuu-Jen Tsai, Kishnani, Priya S., Yin-Hsiu Chien

المصدر: Frontiers in Immunology; 2024, p1-12, 12p

مصطلحات موضوعية: ENZYME replacement therapy, GLYCOGEN storage disease type II, IMMUNOLOGICAL tolerance, TREATMENT effectiveness, LYSOSOMAL storage diseases, THERAPEUTICS

المؤلفون: Kulkarni, Akhil, Chen, Tiffany, Sidransky, Ellen, Han, Tae-Un

المصدر: Genes; Mar2024, Vol. 15 Issue 3, p364, 18p

مصطلحات موضوعية: GAUCHER'S disease, GENE therapy, VIRAL genes, ENZYME replacement therapy, LYSOSOMAL storage diseases, GENETIC vectors, DYSPLASIA

المؤلفون: Hallows, William C.¹ (AUTHOR) william.hallows@codexis.com, Skvorak, Kristen¹ (AUTHOR), Agard, Nick^1,2 (AUTHOR), Kruse, Nikki¹ (AUTHOR), Zhang, Xiyun^1,3 (AUTHOR), Zhu, Yu¹ (AUTHOR), Botham, Rachel C.¹ (AUTHOR), Chng, Chinping¹ (AUTHOR), Shukla, Charu¹ (AUTHOR), Lao, Jessica^1,4 (AUTHOR), Miller, Mathew¹ (AUTHOR), Sero, Antoinette¹ (AUTHOR), Viduya, Judy¹ (AUTHOR), Ismaili, Moulay Hicham Alaoui^1,5 (AUTHOR), McCluskie, Kerryn^1,5 (AUTHOR), Schiffmann, Raphael^6,7 (AUTHOR), Silverman, Adam P.¹ (AUTHOR), Shen, Jin-Song^6,7 (AUTHOR), Huisman, Gjalt W.¹ (AUTHOR)

المصدر: Scientific Reports. 3/23/2023, Vol. 13 Issue 1, p1-16. 16p.

مصطلحات موضوعية: *ANGIOKERATOMA corporis diffusum, *LYSOSOMAL storage diseases, *AMINO acid residues, *GALACTOSIDASES, *THERAPEUTICS, *HEART, *AMINO acid sequence

المؤلفون: Rajan, Deepa S¹ (AUTHOR), Escolar, Maria L¹ (AUTHOR) MLE26@pitt.edu

المصدر: Metabolic Brain Disease. Oct2022, Vol. 37 Issue 7, p2245-2256. 12p.

مصطلحات موضوعية: *LYSOSOMAL storage diseases, *THERAPEUTICS, *GENETIC disorders, *ENZYME replacement therapy, *GENETIC mutation, *LYSOSOMES

المؤلفون: Vest, Ryan T.^1,2, Ching-Chieh Chou³, Hui Zhang², Haney, Michael S.^2,4, Lulin Li⁵, Laqtom, Nouf N.^1,6, Chang, Betty⁵, Shuken, Steven^2,7, Nguyen, Andy⁵, Yerra, Lakshmi⁵, Yang, Andrew C.², Green, Carol⁸, Tanga, Mary⁸, Abu-Remaileh, Monther^1,6, Bassik, Michael C.^6,9 twc@stanford.edu, Frydman, Judith^3,4,9 jfrydman@stanford.edu, Jian Luo⁵ JLuo@pavir.org

المصدر: Proceedings of the National Academy of Sciences of the United States of America. 3/15/2022, Vol. 119 Issue 11, p1-12. 30p.

مصطلحات موضوعية: *SMALL molecules, *LYSOSOMAL storage diseases, *THERAPEUTICS, *PARKINSON'S disease, *INFLAMMATION, *COMMERCIAL products, *ELECTRIC breakdown, *NATURAL products

المؤلفون: Fujii, Takahiro¹ (AUTHOR), Tanaka, Yuta^1,2 (AUTHOR) yuta.tanaka@takeda.com, Oki, Hideyuki³ (AUTHOR), Sato, Sho⁴ (AUTHOR), Shibata, Sachio³ (AUTHOR), Maru, Takamitsu³ (AUTHOR), Tanaka, Maiko¹ (AUTHOR), Onishi, Tomohiro¹ (AUTHOR) tomohiro.onishi@takeda.com

المصدر: Journal of Neurochemistry. Nov2021, Vol. 159 Issue 3, p543-553. 11p.

مصطلحات موضوعية: *GAUCHER'S disease, *LYSOSOMAL storage diseases, *LABORATORY mice, *CENTRAL nervous system, *THERAPEUTICS

المؤلفون: Castelli, Vanessa¹ (AUTHOR) vanessa.castelli@univaq.it, Stamerra, Cosimo Andrea¹ (AUTHOR), d'Angelo, Michele¹ (AUTHOR), Cimini, Annamaria^1,2 (AUTHOR), Ferri, Claudio¹ (AUTHOR) claudio.ferri@cc.univaq.it

المصدر: Clinical Genetics. Sep2021, Vol. 100 Issue 3, p239-247. 9p.

مصطلحات موضوعية: *ANGIOKERATOMA corporis diffusum, *LYSOSOMAL storage diseases, *THERAPEUTICS, *PHENOTYPES, *KIDNEY failure

المؤلفون: van Kuilenburg, André B. P., Hollak, Carla E. M., Travella, Ana, Jacobs, Melisa, Gentilini, Lucas D., Leen, René, der Vlugt, Karen M. M. Ghauharali-van, Stet, Femke S. Beers, Goorden, Susan M. I., van der Veen, Sanne, Criscuolo, Marcelo, Papouchado, Mariana

المصدر: Drugs in R&D; Jun2023, Vol. 23 Issue 2, p141-153, 13p

مصطلحات موضوعية: ANGIOKERATOMA corporis diffusum, THERAPEUTICS, AMINO acid sequence, LYSOSOMAL storage diseases, ENZYME deficiency