Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

1. Download : Download high-res image (108KB)
2. Download : Download full-size image

     

Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells

1. Download : Download high-res image (154KB)
2. Download : Download full-size image

     

Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases

1. Download : Download high-res image (203KB)
2. Download : Download full-size image

     

Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

1. Download : Download high-res image (114KB)
2. Download : Download full-size image

     

Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques

1. Download : Download high-res image (131KB)
2. Download : Download full-size image

     

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

1. Download : Download high-res image (188KB)
2. Download : Download full-size image

     

Dual-AAV delivering split prime editor system for in vivo genome editing

1. Download : Download high-res image (87KB)
2. Download : Download full-size image

     

Chemogenetics as a neuromodulatory approach to treating neuropsychiatric diseases and disorders

1. Download : Download high-res image (137KB)
2. Download : Download full-size image

     

Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor

1. Download : Download high-res image (94KB)
2. Download : Download full-size image

     

Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells

1. Download : Download high-res image (208KB)
2. Download : Download full-size image

     

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9

1. Download : Download high-res image (149KB)
2. Download : Download full-size image

     

Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase

1. Download : Download high-res image (145KB)
2. Download : Download full-size image

     

AAV vectors: The Rubik’s cube of human gene therapy

1. Download : Download high-res image (386KB)
2. Download : Download full-size image

     

Targeting the hepatitis B cccDNA with a sequence-specific ARCUS nuclease to eliminate hepatitis B virus in vivo

1. Download : Download high-res image (141KB)
2. Download : Download full-size image

     

Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F

1. Download : Download high-res image (174KB)
2. Download : Download full-size image

     

Gene-Loaded Nanoparticle-Coated Sutures Provide Effective Gene Delivery to Enhance Tendon Healing

1. Download : Download high-res image (153KB)
2. Download : Download full-size image

     

IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification

1. Download : Download high-res image (129KB)
2. Download : Download full-size image

     

CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genes

1. Download : Download high-res image (203KB)
2. Download : Download full-size image

     

Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction

1. Download : Download high-res image (101KB)
2. Download : Download full-size image

     

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

1. Download : Download high-res image (55KB)
2. Download : Download full-size image