Home > Resources > Videos > SMA Type 1: How Gene Therapy Works

SMA Type 1: How Gene Therapy Works

A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published Nov. 1, 2017 in the New England Journal of Medicine. This animation describes how the process works.

Spotlight

Aker BioMarine AS

Aker BioMarine is a biotech innovator and Antarctic krill -harvesting company, dedicated to improving human and planetary health. The company develops krill-based ingredients for nutraceutical, aquaculture, and animal feed applications. The company’s fully transparent value chain stretches from sustainable krill harvesting in pristine Antarctic waters through its Montevideo logistics hub, Houston production plant, and all the way to customers around the world.

OTHER VIDEOS

Image analysis software for true ultra high content data on the MACSima™ Imaging System

video | July 28, 2023

Qi Tissue Image Analysis Software for the MACSima™ Imaging Platform is the key to unlocking staggering analytical ability with ultra high content imaging data generated on the revolutionary new MACSima Imaging Platform....

Watch Now

Bioprinting Living Cells: The Next Frontier in Biotechnology Technology

video | July 30, 2023

Bioprinting with living human cells is transforming healthcare as we know it. From Slurpee-like substances to molding 3D tissues, explore the cutting-edge world of 3D bioprinting. Dr. Mark Skylar-Scott from Stanford University discusses the fascinating realm of 3D bioprinting, a groundbreaking approach that goes beyond traditional plastic printing....

Watch Now

VIA Thaw™ automated dry thawer

video | May 9, 2023

VIA Thaw™ dry automated thawers give you the confidence to reliably recover cryopreserved cellular products. VIA Thaw™ L1000 intended for research or manufacturing use only is an automated dry thawer that you can use for controlled, consistent thawing of cellular products in cryobags....

Watch Now

NeatCell C-Pro application software for the Sepax™ C-Pro instrument

video | May 9, 2023

Meet the NeatCell C-Pro application. It automates the mononuclear cell fraction enrichment from diverse cellular products, and is designed for use with a density-gradient medium like Ficoll-Paque media with the Sepax C-Pro instrument and CT-90.1 single-use kit....

Watch Now

Image analysis software for true ultra high content data on the MACSima™ Imaging System

video | July 28, 2023

Qi Tissue Image Analysis Software for the MACSima™ Imaging Platform is the key to unlocking staggering analytical ability with ultra high content imaging data generated on the revolutionary new MACSima Imaging Platform....

Watch Now

Bioprinting Living Cells: The Next Frontier in Biotechnology Technology

video | July 30, 2023

Bioprinting with living human cells is transforming healthcare as we know it. From Slurpee-like substances to molding 3D tissues, explore the cutting-edge world of 3D bioprinting. Dr. Mark Skylar-Scott from Stanford University discusses the fascinating realm of 3D bioprinting, a groundbreaking approach that goes beyond traditional plastic printing....

Watch Now

VIA Thaw™ automated dry thawer

video | May 9, 2023

VIA Thaw™ dry automated thawers give you the confidence to reliably recover cryopreserved cellular products. VIA Thaw™ L1000 intended for research or manufacturing use only is an automated dry thawer that you can use for controlled, consistent thawing of cellular products in cryobags....

Watch Now

NeatCell C-Pro application software for the Sepax™ C-Pro instrument

video | May 9, 2023

Meet the NeatCell C-Pro application. It automates the mononuclear cell fraction enrichment from diverse cellular products, and is designed for use with a density-gradient medium like Ficoll-Paque media with the Sepax C-Pro instrument and CT-90.1 single-use kit....

Watch Now
MORE VIDEOS

Spotlight

Aker BioMarine AS

Aker BioMarine is a biotech innovator and Antarctic krill -harvesting company, dedicated to improving human and planetary health. The company develops krill-based ingredients for nutraceutical, aquaculture, and animal feed applications. The company’s fully transparent value chain stretches from sustainable krill harvesting in pristine Antarctic waters through its Montevideo logistics hub, Houston production plant, and all the way to customers around the world.

Events