Search | Virtual Health Library

1.

Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches.

Leal, Andrés Felipe; Herreno-Pachón, Angelica María; Benincore-Flórez, Eliana; Karunathilaka, Amali; Tomatsu, Shunji.

Int J Mol Sci ; 25(5)2024 Feb 20.

Article in English | MEDLINE | ID: mdl-38473704

2.

Molecular Mechanisms in Pathophysiology of Mucopolysaccharidosis and Prospects for Innovative Therapy.

Ago, Yasuhiko; Rintz, Estera; Musini, Krishna Sai; Ma, Zhengyu; Tomatsu, Shunji.

Int J Mol Sci ; 25(2)2024 Jan 17.

Article in English | MEDLINE | ID: mdl-38256186

3.

Morquio A Syndrome: Identification of Differential Patterns of Molecular Pathway Interactions in Bone Lesions.

Álvarez, J Victor; Bravo, Susana B; Chantada-Vázquez, María Pilar; Pena, Carmen; Colón, Cristóbal; Tomatsu, Shunji; Otero-Espinar, Francisco J; Couce, María L.

Int J Mol Sci ; 25(6)2024 Mar 12.

Article in English | MEDLINE | ID: mdl-38542208

4.

The landscape of CRISPR/Cas9 for inborn errors of metabolism.

Leal, Andrés Felipe; Fnu, Nidhi; Benincore-Flórez, Eliana; Herreño-Pachón, Angelica María; Echeverri-Peña, Olga Yaneth; Alméciga-Díaz, Carlos Javier; Tomatsu, Shunji.

Mol Genet Metab ; 138(1): 106968, 2023 01.

Article in English | MEDLINE | ID: mdl-36525790

5.

Molecular Trojan Horses for treating lysosomal storage diseases.

Leal, Andrés Felipe; Inci, Orhan Kerim; Seyrantepe, Volkan; Rintz, Estera; Celik, Betul; Ago, Yasuhiko; León, Daniel; Suarez, Diego A; Alméciga-Díaz, Carlos Javier; Tomatsu, Shunji.

Mol Genet Metab ; 140(3): 107648, 2023 11.

Article in English | MEDLINE | ID: mdl-37598508

6.

Tracheal Narrowing and Its Impact on Anesthesia Care in Patients With Morquio A (Mucopolysaccharidosis Type IVA): An Observational Study.

Theroux, Mary C; DiCindio, Sabina; Averill, Lauren W; Pizarro, Christian; Oommen, Abraham; Bober, Michael B; Ditro, Colleen; Campbell, Jeffrey; Duker, Angela L; Jones, Taylor; Passi, Vandna; Barth, Patrick; Schmidt, Richard J; Little, Mary; Mackenzie, Stuart; Tomatsu, Shunji; Mackenzie, William G.

Anesth Analg ; 137(5): 1075-1083, 2023 11 01.

Article in English | MEDLINE | ID: mdl-37862598

7.

Mucopolysaccharidosis IVA: Current Disease Models and Drawbacks.

Leal, Andrés Felipe; Alméciga-Díaz, Carlos Javier; Tomatsu, Shunji.

Int J Mol Sci ; 24(22)2023 Nov 09.

Article in English | MEDLINE | ID: mdl-38003337

8.

Bone Growth Induction in Mucopolysaccharidosis IVA Mouse.

Rintz, Estera; Herreño-Pachón, Angélica María; Celik, Betul; Nidhi, Fnu; Khan, Shaukat; Benincore-Flórez, Eliana; Tomatsu, Shunji.

Int J Mol Sci ; 24(12)2023 Jun 08.

Article in English | MEDLINE | ID: mdl-37373036

9.

Molecular Mechanism of Induction of Bone Growth by the C-Type Natriuretic Peptide.

Rintz, Estera; Wegrzyn, Grzegorz; Fujii, Toshihito; Tomatsu, Shunji.

Int J Mol Sci ; 23(11)2022 May 25.

Article in English | MEDLINE | ID: mdl-35682595

10.

Regulation of Molecular Targets in Osteosarcoma Treatment.

Celik, Betul; Cicek, Kader; Leal, Andrés Felipe; Tomatsu, Shunji.

Int J Mol Sci ; 23(20)2022 Oct 20.

Article in English | MEDLINE | ID: mdl-36293439

11.

Mucopolysaccharidoses: Cellular Consequences of Glycosaminoglycans Accumulation and Potential Targets.

Leal, Andrés Felipe; Benincore-Flórez, Eliana; Rintz, Estera; Herreño-Pachón, Angélica María; Celik, Betul; Ago, Yasuhiko; Alméciga-Díaz, Carlos Javier; Tomatsu, Shunji.

Int J Mol Sci ; 24(1)2022 Dec 28.

Article in English | MEDLINE | ID: mdl-36613919

12.

Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy.

Piechnik, Matthew; Amendum, Paige C; Sawamoto, Kazuki; Stapleton, Molly; Khan, Shaukat; Fnu, Nidhi; Álvarez, Victor; Pachon, Angelica Maria Herreño; Danos, Olivier; Bruder, Joseph T; Karumuthil-Melethil, Subha; Tomatsu, Shunji.

Int J Mol Sci ; 23(20)2022 Oct 21.

Article in English | MEDLINE | ID: mdl-36293546

13.

Tracheal narrowing in children and adults with mucopolysaccharidosis type IVA: evaluation with computed tomography angiography.

Averill, Lauren W; Kecskemethy, Heidi H; Theroux, Mary C; Mackenzie, William G; Pizarro, Christian; Bober, Michael B; Ditro, Colleen P; Tomatsu, Shunji.

Pediatr Radiol ; 51(7): 1202-1213, 2021 Jun.

Article in English | MEDLINE | ID: mdl-33608742

14.

Plasma Proteomic Analysis in Morquio A Disease.

Álvarez, José V; Bravo, Susana B; Chantada-Vázquez, María Pilar; Barbosa-Gouveia, Sofía; Colón, Cristóbal; López-Suarez, Olalla; Tomatsu, Shunji; Otero-Espinar, Francisco J; Couce, María L.

Int J Mol Sci ; 22(11)2021 Jun 07.

Article in English | MEDLINE | ID: mdl-34200496

15.

Advances in glycosaminoglycan detection.

Khan, Shaukat A; Mason, Robert W; Kobayashi, Hironori; Yamaguchi, Seiji; Tomatsu, Shunji.

Mol Genet Metab ; 130(2): 101-109, 2020 06.

Article in English | MEDLINE | ID: mdl-32247585

16.

Neonatal combination therapy improves some of the clinical manifestations in the Mucopolysaccharidosis type I murine model.

Santi, Ludovica; De Ponti, Giada; Dina, Giorgia; Pievani, Alice; Corsi, Alessandro; Riminucci, Mara; Khan, Shaukat; Sawamoto, Kazuki; Antolini, Laura; Gregori, Silvia; Annoni, Andrea; Biondi, Andrea; Quattrini, Angelo; Tomatsu, Shunji; Serafini, Marta.

Mol Genet Metab ; 130(3): 197-208, 2020 07.

Article in English | MEDLINE | ID: mdl-32439268

17.

Newborn screening of mucopolysaccharidoses: past, present, and future.

Arunkumar, Nivethitha; Langan, Thomas J; Stapleton, Molly; Kubaski, Francyne; Mason, Robert W; Singh, Rajendra; Kobayashi, Hironori; Yamaguchi, Seiji; Suzuki, Yasuyuki; Orii, Kenji; Orii, Tadao; Fukao, Toshiyuki; Tomatsu, Shunji.

J Hum Genet ; 65(7): 557-567, 2020 Jul.

Article in English | MEDLINE | ID: mdl-32277174

18.

Bone-Specific Drug Delivery for Osteoporosis and Rare Skeletal Disorders.

Sawamoto, Kazuki; Álvarez, J Víctor; Herreño, Angélica María; Otero-Espinar, Francisco J; Couce, Maria L; Alméciga-Díaz, Carlos J; Tomatsu, Shunji.

Curr Osteoporos Rep ; 18(5): 515-525, 2020 10.

Article in English | MEDLINE | ID: mdl-32845464

19.

Evading the AAV Immune Response in Mucopolysaccharidoses.

Piechnik, Matthew; Sawamoto, Kazuki; Ohnishi, Hidenori; Kawamoto, Norio; Ago, Yasuhiko; Tomatsu, Shunji.

Int J Mol Sci ; 21(10)2020 May 13.

Article in English | MEDLINE | ID: mdl-32414007

20.

Mucopolysaccharidosis IVA: Diagnosis, Treatment, and Management.

Sawamoto, Kazuki; Álvarez González, José Víctor; Piechnik, Matthew; Otero, Francisco J; Couce, Maria L; Suzuki, Yasuyuki; Tomatsu, Shunji.

Int J Mol Sci ; 21(4)2020 Feb 23.

Article in English | MEDLINE | ID: mdl-32102177

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