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Gene Therapy

Tan RNA strands float against a blue background

New Discovery Enables Gene Therapy for Muscular Dystrophies, Other Disorders

byUniversity of Rochester Medical Center

StitchR delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models

| 4 min read

Laboratory technicians using bench meters to measure pH levels in a quality control lab setting

The Importance of pH Measurement in Cell Culture Research

byGayle Gleichauf

| 3 min read

Setting New Standards for Precision in Cell and Gene Therapy

Enhance the Accuracy of Viral Titer Quantification with Droplet Digital PCR

byBio-Rad LaboratoriesandLab Manager

Updated | 2 min read

3D render of white DNA double helix against a blue background

Gene Therapy Helps Patients with a Rare Pediatric Brain Disease

byMass General Brigham

First gene therapy approved for cerebral adrenoleukodystrophy shows promising results

| 4 min read

A section of a double helix is removed and replaced

Single-Dose Gene Therapy Is Potentially Life-Changing for Adults with Hemophilia B

byUniversity of Pennsylvania School of Medicine

Study results formed the basis of the therapy's FDA approval in April 2024

| 3 min read

Macro shot of a person's eye

New Gene Therapy for Treating Glaucoma

byTrinity College Dublin

The treatment may also address age-related macular degeneration

| 2 min read

a 3d illustration of a DNA molecule with one strand in blue and the other in red. Part of the red strand has been separated from the rest of the molecule. The strands are composed of many small lines connecting to dots with additional glowing dots surrounding the molecule, all on a dark blue background.

New Gene Therapy Treatment Restores Vision in Patients with Genetic Blindness

byUniversity of Florida

Those with certain genetic blindness saw a 10K-fold improve in light sensitivity

| 3 min read

a 3d illustration of a DNA molecule with one strand in blue and the other in red. Part of the red strand has been separated from the rest of the molecule.

‘Faster, Safer, and More Efficient’ Gene Editing

byJohn A. Burns School of Medicine, University of Hawi’i Manoa

This new advancement could open the door to treatments for hundreds of conditions

| 2 min read

a photo representing hearing, showing a man's ear with his hand behind it, overlayed with a circular target and audiowaveforms

With Gene Editing, Mice with a Form of Inherited Deafness Can Hear Again

byNational Center for Advancing Translational Sciences

Approach restores hearing in adult mice, opening new possibilities for treating genetic conditions in people

| 3 min read

Lab staff working in lab

Industry Expertise Improves Capsid Separation

byLab ManagerandCytiva

Preliminary testing and collaboration can combat the many pitfalls of scaling up viral vector production

Updated | 5+ min read

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