Sangamo showcases progress in our pre-clinical programs at ASGCT.

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Genome Editing

Resolution of hepatic fibrosis after zinc-finger nuclease-mediated gene editing in the PiZ mouse model of human alpha-1-antitrypsin deficiency liver disease
March 7th, 2023
Anion-Exchange HPLC Assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-Associated Virus Serotypes
April 23rd, 2021
ZFN-Mediated In Vivo Gene Editing in Hepatocytes Leads to Supraphysiologic α-Gal A Activity and Effective Substrate Reduction in Fabry Mice
March 26th, 2021
Highly Efficient Gene Editing of Cystic Fibrosis Patient-derived Airway Basal Cells Results in Functional CFTR Correction
May 7th, 2020
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
May 7th, 2020
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
May 7th, 2020
In vivo genome editing restores haemostasis in a mouse model of haemophilia.
August 24th, 2018
An improved zinc-finger nuclease architecture for highly specific genome editing.
August 24th, 2018
Highly efficient endogenous human gene correction using designed zinc-finger nucleases.
August 24th, 2018
Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
August 24th, 2018