Global epigenetic regulation and capsid delivery license agreement with Genentech
Click here to view
Careers
Core Values
Diversity, Equity and Inclusion
Open Positions
Employee Benefits
Clinical Trials
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Modular Integrase (MINT) Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Modular Integrase (MINT) Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
LinkedIn
Twitter
Technology
Anion-Exchange HPLC Assay for Separation and Quantification of Empty and Full Capsids in Multiple Adeno-Associated Virus Serotypes
April 23rd, 2021
Axonal transport of AAV9 in nonhuman primate brain.
August 24th, 2018
Functional footprinting of regulatory DNA.
August 24th, 2018
Highly efficient homology-driven genome editing in human T cells by combining zinc-finger nuclease mRNA and AAV6 donor delivery.
August 24th, 2018
Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.
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
Zinc-finger protein-targeted gene regulation: genomewide single-gene specificity.
August 24th, 2018
Genome editing with engineered zinc finger nucleases.
August 24th, 2018