Gene Therapy

Supporting start-ups to accelerate biomedical innovations 1:32
Supporting start-ups to accelerate biomedical innovations
Kurt Gielen • 17 Nov 2021
Advanced therapies for retinitis pigmentosa 2:27
Advanced therapies for retinitis pigmentosa
Gil Carrasquinho • 2 Jul 2021
Challenges with translating gene therapies for neurodevelopmental disorders 2:44
Challenges with translating gene therapies for neurodevelopmental disorders
Ralf Schmid • 16 Nov 2021
Non-viral gene delivery & gene editing: highlights from ESGCT 2:56
Non-viral gene delivery & gene editing: highlights from ESGCT
Ralf Schmid • 16 Nov 2021
RP-L301 lentiviral gene therapy for PKD 3:47
RP-L301 lentiviral gene therapy for PKD
Jose-Luis López-Lorenzo • 15 Nov 2021
An update on the Phase I trial evaluating RP-L301 in PKD 7:24
An update on the Phase I trial evaluating RP-L301 in PKD
Jose-Luis López-Lorenzo • 15 Nov 2021
Gene therapy for PKD: addressing the unmet clinical need 3:50
Gene therapy for PKD: addressing the unmet clinical need
Jose-Luis López-Lorenzo • 15 Nov 2021
Highlights from ESGCT 2021: gene therapy clinical data, advances in gene editing & AAV toxicity 3:27
Highlights from ESGCT 2021: gene therapy clinical data, advances in gene editing & AAV toxicity
Paul Wuh-Liang Hwu • 17 Nov 2021
Eladocagene exuparvovec: AAV2-mediated gene therapy for AADC deficiency 8:51
Eladocagene exuparvovec: AAV2-mediated gene therapy for AADC deficiency
Paul Wuh-Liang Hwu • 17 Nov 2021
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 gene-replacement therapy landscape for CDKL5-deficiency disorder 3:49
The gene-replacement therapy landscape for CDKL5-deficiency disorder
Ralf Schmid • 16 Nov 2021
AAV9-mediated gene therapy for CDKL5-deficiency disorder 4:23
AAV9-mediated gene therapy for CDKL5-deficiency disorder
Ralf Schmid • 16 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
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