Metabolic Disorder

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