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X
Paul Gissen
377  Ergebnisse:
Personensuche X
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1

Cerliponase alfa for the treatment of CLN2 disease in a pat..:

Schulz, Angela ; de los Reyes, Emily C. ; Specchio, Nicola...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 108034 , 2024
Link: https://doi.org/10.1016/..
 
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2

Generation of induced pluripotent stem cells (UCLi024-A) fr..:

Duff, Claire ; Islam, Madeha ; Gagliano, Onelia...
Stem Cell Research.  76 (2024)  - p. 103365 , 2024
Link: https://doi.org/10.1016/..
 
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3

Exploring concurrent validity of the CLN2 Clinical Rating S..:

Specchio, Nicola ; Gissen, Paul ; de los Reyes, Emily...
PLOS ONE.  19 (2024)  5 - p. e0302382 , 2024
Link: https://doi.org/10.1371/..
 
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4

Characterization of two human induced pluripotent stem cell..:

Ofrim, Marisa ; Little, Daniel ; Nazari, Mina...
Stem Cell Research.  74 (2024)  - p. 103291 , 2024
Link: https://doi.org/10.1016/..
 
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5

RGX-381: Interim results from the first-in-human clinical t..:

Ohnsman, Christina ; Bailey, Alexander M. ; Huang, Wei...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107987 , 2024
Link: https://doi.org/10.1016/..
 
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6

Liver‐directed gene therapy for inherited metabolic disease:

Baruteau, Julien ; Brunetti‐Pierri, Nicola ; Gissen, Paul
Journal of Inherited Metabolic Disease.  47 (2024)  1 - p. 9-21 , 2024
Link: https://doi.org/10.1002/..
 
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7

Results of a phase III, randomized, placebo-controlled cros..:

Fields, Taylor ; Bremova-Ertl, Tatiana ; Ramaswami, Uma...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 107827 , 2024
Link: https://doi.org/10.1016/..
 
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8

Evolution of Movement Disorders in Patients With CLN2-Batte..:

Spaull, Robert ; Soo, Audrey K. ; Batzios, Spyros...
Neurology.  103 (2024)  3 - p. , 2024
Link: https://doi.org/10.1212/..
 
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9

Safety and efficacy of cerliponase alfa in children with ne..:

Schulz, Angela ; Specchio, Nicola ; de los Reyes, Emily...
The Lancet Neurology.  23 (2024)  1 - p. 60-70 , 2024
Link: https://doi.org/10.1016/..
 
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10

Buffy Coat Score as a Biomarker of Treatment Response in Ne..:

Sivananthan, Siyamini ; Lee, Laura ; Anderson, Glenn...
Brain Sciences.  13 (2023)  2 - p. 209 , 2023
Link: https://doi.org/10.3390/..
 
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11

Genetic Therapy Approaches for Ornithine Transcarbamylase D..:

Seker Yilmaz, Berna ; Gissen, Paul
Biomedicines.  11 (2023)  8 - p. 2227 , 2023
Link: https://doi.org/10.3390/..
 
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12

Features of Congenital Arthrogryposis Due to Abnormalities ..:

Picker, Sarah MacKenzie ; Parker, George ; Gissen, Paul
International Journal of Molecular Sciences.  24 (2023)  17 - p. 13545 , 2023
Link: https://doi.org/10.3390/..
 
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13

New Perspectives in Dried Blood Spot Biomarkers for Lysosom..:

Spiewak, Justyna ; Doykov, Ivan ; Papandreou, Apostolos...
International Journal of Molecular Sciences.  24 (2023)  12 - p. 10177 , 2023
Link: https://doi.org/10.3390/..
 
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14

Fetal gene therapy:

Waddington, Simon N. ; Peranteau, William H. ; Rahim, Ahad A....
Journal of Inherited Metabolic Disease.  47 (2023)  1 - p. 192-210 , 2023
Link: https://doi.org/10.1002/..
 
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15

Metachromatic leukodystrophy: A single centre review of fea..:

Sivananthan, Siyamini ; Davison, James ; Chakrapani, Anupam.
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107321 , 2023
Link: https://doi.org/10.1016/..
 
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