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Metabolic Disorder
1:58
Treating Krabbe disease with FBX-101
John Maslowski
• 27 Jan 2022
6:19
LYS-GM101 AAV gene therapy for GM1 gangliosidosis
Michael Hocquemiller
• 15 Nov 2021
3:42
GNT0003: AAV gene therapy for Crigler-Najjar syndrome
Lorenzo D'Antiga
• 18 Nov 2021
4:31
The safety of AVR-RD-01 gene therapy for Fabry disease
Mark Thomas
• 29 Oct 2021
2:31
The unmet medical need for Crigler-Najjar syndrome
Lorenzo D'Antiga
• 18 Nov 2021
3:08
Educating HCPs and patients on novel therapies for metabolic disorders
Mark Thomas
• 29 Oct 2021
2:38
LYS-SAF302 AAV gene therapy for MPS IIIA
Michael Hocquemiller
• 15 Nov 2021
1:16
Overcoming immunogenicity of gene therapies
Timothy Miller
• 18 Nov 2021
0:59
RESKUE: AAV-based gene therapy for Krabbe disease
Timothy Miller
• 18 Nov 2021
2:07
Treating Krabbe disease with AAV-based gene therapies
Timothy Miller
• 18 Nov 2021
3:22
The role of regenerative medicine in treating diabetes
Lorenzo Piemonti
• 11 Nov 2021
4:31
The future of regenerative medicine in transplantation
Giuseppe Orlando
• 11 Nov 2021
4:36
Bio-engineering vascularized islet organs for T1D
Lorenzo Piemonti
• 11 Nov 2021
3:34
Challenges in β cell replacement therapy
Lorenzo Piemonti
• 11 Nov 2021
1:57
Tailored nucleases for primary hyperoxaluria
Devyn Smith
• 29 Oct 2021
2:34
Advanced cell selection for the manufacture of ProTrans cell therapy
Mathias Svahn
• 6 Oct 2021
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