Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

Tardieu, Marc, Zérah, Michel, Husson, Béatrice, de Bournonville, Stéphanie, Deiva, Kumaran, Adamsbaum, Catherine, Vincent, Fanny, Hocquemiller, Michael +9 more·2014·Human Gene Therapy
DOI 10.1089/hum.2013.238

Cited by (261)

Items 201–261 of 261

  1. Validation of Anti-Adeno Associated Virus Serotype rh10 (AAVrh.10) Total and Neutralizing Antibody Immunogenicity Assays
    Elizabeth Butala, Thien Nguyen, Nithya Selvan, Luke Armstrong, Michelle M. Miller, Lynn A. Kamen, Todd R. Lester, Roman P. Wernyj, Richie Khanna, Jim McNally et al. · 2023 · Pharmaceutical Research
  2. Current and Emerging Therapies for Lysosomal Storage Disorders
    Diego Agustín Abelleyra Lastoria, Sophie Keynes, Derralynn A. Hughes · 2025 · Drugs
  3. NGF and BDNF Gene Therapy for Alzheimer’s Disease
    Mark H. Tuszynski, Alan H. Nagahara · 2016 · Springer eBooks
  4. Innovative Treatments for Mucopolysaccharidoses
    Florian B. Lagler · 2018 · Journal of Child Science
  5. Preclinical use of a clinically-relevant scAAV9/SUMF1 vector for the treatment of multiple sulfatase deficiency
    Maximiliano F Presa, Rachel M. Bailey, Somdatta Ray, Lauren Bailey, Saurabh Tata, Tara C. Murphy, Pierre-Alexandre Piec, Harold Combs, Steven James Gray, Cathleen M. Lutz · 2025 · Communications Medicine
  6. Fetal gene therapy for neurodegenerative lysosomal storage diseases
    Julien Baruteau, Simon N. Waddington · 2019 · Journal of Inherited Metabolic Disease
  7. Incidence, Timing and Clinical Significance of Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis
    Niccolò Maurizi, Enrico Ammirati, Elizabeth Silver, Kimberly N. Hong, Quan M. Bui, Alessia Argirò, Iacopo Olivotto, Eric Adler · 2026 · Circulation Heart Failure
  8. Mi hija, mi vida / tu vivras ma fille (2018) de Gabriel Aghion, de la medicina familiar a la biotecnología
    Gabriela Cevallos-Solorzano, Vicente Paúl Villamagua León, Renata Aguirre-Fierro, Jennifer Estefanía Jaramillo Suquilanda, Kevin Sebastián Chicaiza González, Dominique Anahí Bravo Pinzón, Natalia Bailón-Moscoso · 2022 · Revista de Medicina y Cine
  9. Production and Application of Multicistronic Constructs for Various Human Disease Therapies
    Alisa A. Shaimardonova, Daria S. Chulpanova, Kristina Viktorovna Kitaeva, Ilmira I. Abdrakhmanova, Vladislav M. Chernov, Catrin Sian Rutland, Albert Anatolyevich Rizvanov, Valeriya V. Solovyeva · 2021
  10. Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways
    Margareta Rybarikova, Amanda Almacellas‐Barbanoj, Stéphanie Schorge, Nicole Déglon · 2022 · Human Gene Therapy
  11. Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction
    Jenna Magat, Samantha Jones, Brian Baridon, Vishal Agrawal, Hio Wong, Alexander Giaramita, Linley Mangini, Britta Handyside, Catherine Vitelli, Monica Parker et al. · 2022 · Journal of Biological Chemistry
  12. Molecular environment and atypical function: What do we know about enzymes associated with Mucopolysaccharidoses?
    Weijing Kong, Cheng Lu, Yingxue Ding, Yan Meng · 2022 · Orphanet Journal of Rare Diseases
  13. The Mucopolysaccharidoses: Prenatal Diagnosis, Neonatal Screening and Emerging Therapies
    Lorne A. Clarke · 2021 · Wiley eBooks
  14. Ocular gene therapy mediated by AAV vectors: an overview of immunosuppressive treatments and immunomonitoring of patients involved in clinical trials
    Duohao Ren, Gaëlle Anne Chauveau, Emilie Cabon, Julie Vendomèle, Catherine Vignal‐Clermont, Hanadi Saliba, Divya Ail, Deniz Dalkara, Sylvain Fisson · 2026 · Progress in Retinal and Eye Research
  15. The top 100 most cited articles on mucopolysaccharidoses: a bibliometric analysis
    Ruyu Liao, Rongrong Geng, Yue Yang, Yufan Xue, Lili Chen, Lan Chen · 2024 · Frontiers in Genetics
  16. Human Neuraminidase 3 reduces the severity of Tay-Sachs disease symptoms in a mouse model
    Hande Basırlı, Volkan Seyrantepe · 2026 · Neurochemistry International
  17. Lysosomale Speicherkrankheiten: Therapeutische Optionen
    Michael Beck · 2015 · Medizinische Genetik
  18. Enzyme replacement and gene therapy for mucopolysaccharidoses: current progress and future directions
    Michael Beck · 2015 · Expert Opinion on Orphan Drugs
  19. Gene therapy approaches in central nervous system regenerative medicine
    Assumpció Bosch, Miguel Chillón · 2020 · Elsevier eBooks
  20. Gene therapy
    Stefan Worgall, Ronald G. Crystal · 2020 · Elsevier eBooks
  21. Efficient transduction of adeno-associated virus vectors into gerbil hippocampus with an appropriate combination of viral capsids and promoters
    Yoshihide Sehara, Kuniko Shimazaki, Fumio Kurosaki, Naoki Kaneko, Ryosuke Uchibori, Masashi Urabe, Kensuke Kawai, Hiroaki Mizukami · 2018 · Neuroscience Letters
  22. Recent progress in gene therapies for mucopolysaccharidoses
    Virginia Maria Ginocchio, Nicola Brunetti‐Pierri · 2018 · Expert Opinion on Orphan Drugs
  23. A Rare case of Sanfilippo syndrome type “C”
    Ansh Agarwal, Kashish Goyal, Priyanshu Mathur, Avisha Mathur · 2020 · Indian Journal of Child Health
  24. Gene Therapy Applications
    Clévio Nóbrega, Liliana S. Mendonça, Carlos A. Matos · 2020 · Springer eBooks
  25. Les thérapies géniques en neurologie
    J.-B. Brunet de Courssou, Kumaran Deiva · 2023 · Pratique Neurologique - FMC
  26. Transduction catalysis: doxorubicin accelerates and enhances rAAV-mediated gene expression in the cortex of mouse, cat and monkey
    Hongliang Gong, Nini Yuan, Zhiming Shen, Cheng Tang, Stewart D. Shipp, Liling Qian, Yiliang Lu, Ian Max Andolina, Shenghai Zhang, Jihong Wu et al. · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  27. Lysosomal Diseases
    Timothy Martin Cox · 2015 · Elsevier eBooks
  28. Prenatal Diagnosis of the Mucopolysaccharidoses and Postnatal Enzyme Replacement Therapy
    John J. Hopwood · 2015 · Wiley eBooks
  29. Prospective therapies for mucopolysaccharidoses
    Anna Tylki‐Szymańska, Agnieszka Jurecka · 2015 · Expert Opinion on Orphan Drugs
  30. Entwicklung neuer Therapien für seltene Erkrankungen
    Julia B. Hennermann · 2017 · Monatsschrift Kinderheilkunde
  31. Intracerebral gene therapy for mucopolysaccharidosis type IIIB syndrome
    Kim M. Hemsley, Adeline A. Lau · 2017 · The Lancet Neurology
  32. Intravenously administered gene therapy for neuronopathic Gaucher disease
    Giulia Massaro · 2018 · UCL Discovery (University College London)
  33. The Neurogenic potential of stem cells is altered in Mucopolysaccharidosis type IIIA
    Rebecca J. Lehmann · 2019 · Adelaide Research & Scholarship (AR&S) (University of Adelaide)
  34. Lysosomal disease
    Patrick B. Deegan, Timothy Martin Cox · 2020
  35. Multiple Enzyme Deficiencies
    Nicole Muschol, Cornelia Rudolph, Thomas Braulke, Nicola Brunetti‐Pierri, Graciana Diez‐Roux, Andrea Ballabio · 2022 · Wiley eBooks
  36. Lysosomal Diseases
    Timothy Martin Cox · 2022 · Elsevier eBooks
  37. Additional file 2 of Is the eye a window to the brain in Sanfilippo syndrome?
    Helen Beard, Glyn Chidlow, Neumann, Daniel, Nazzmer Nazri, Meghan L. Douglass, Paul J. Trim, Marten F. Snel, Robert James Casson, Kim M. Hemsley · 2020 · Figshare
  38. Additional file 11 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  39. Additional file 9 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  40. Additional file 4 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  41. Additional file 13 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  42. Additional file 12 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  43. Additional file 8 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  44. Additional file 6 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  45. Additional file 3 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  46. Additional file 10 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  47. Additional file 7 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  48. Additional file 5 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  49. Additional file 1 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  50. Additional file 1 of Is the eye a window to the brain in Sanfilippo syndrome?
    Helen Beard, Glyn Chidlow, Neumann, Daniel, Nazzmer Nazri, Meghan L. Douglass, Paul J. Trim, Marten F. Snel, Robert James Casson, Kim M. Hemsley · 2020 · Figshare
  51. Additional file 2 of Intraparenchymal convection enhanced delivery of AAV in sheep to treat Mucopolysaccharidosis IIIC
    Claire O’Leary, Gabriella M. A. Forte, Mitchell, Nadia L., Amir Saam Youshani, A. Stephens Dyer, Martin P. Wellby, Katharina N. Russell, Murray, Samantha J., Nelly Jolinon, Simon Allan Jones et al. · 2024 · Figshare
  52. Additional file 1 of Brain-targeted delivery of neuroprotective survival gene minimizing hematopoietic cell contamination: implications for Parkinson’s disease treatment
    Min Hak Lee, Sukyeong Kang, Um, Ki-Hwan, Seok Won Lee, Hyorin Hwang, Baek, Kyunghwa, Jin Woo Choi · 2024 · Figshare
  53. Recent Therapies Including Gene Therapy and Immunotherapy in Overcoming the Gaps in the Treatment of Neurodegeneration
    Sakshi Goswami, Yeshvandra Verma, Suresh Vir Singh Rana · 2026 · Springer eBooks
  54. Optimized AAVrh10 and Factor 9 vectors demonstrate improved phenotypic rescue in hemophilia B mice
    Shamshul Huda, Mohankumar B. Senthilkumar, Dikshika Bihani, Pratiksha Sarangi, G. Narendra Kumar, Dinesh Chandra, Giridhara Rao Jayandharan · 2026 · Thrombosis Research
  55. Comprendre les mécanismes cellulaires déficients dans la MPS VII par l'utilisation de neurones humains dérivés d'iPSC.
    Sophie Creyssels · 2015
  56. Développement préclinique d'une thérapie génique pro-angiogénique combinée de l'ischémie cardiaque et du membre inférieur
    Edith Renaud-Gabardos · 2016 · theses.fr (ABES)
  57. Matrices tridimensionnelles issues de la chimie verte pour le traitement de l’insuffisance cardiaque par thérapie cellulaire-génique
    Raya Bushkalova · 2016
  58. Crossing the blood–brain barrier: Advances and frontiers of AAV gene therapy in central nervous system disorders
    Wenjian Han, Zi-Long QIU · 2026 · Shengming kexue
  59. Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS)
    Ezekiel S. Oloruntimehin, Alexander S. Malogolovkin · 2026 · International Journal of Molecular Sciences
  60. Revolutionizing Neuroprotection
    Ahana Hazra, Rideb Chakraborty, Naureen Afrose, Pratibha Bhowmick, Mithun Bhowmick · 2026 · Wiley eBooks
  61. Mucopolysaccharidosis Type III (MPS III)
    2026 · Elsevier eBooks