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Conceptual gene therapy illustration with 3D multicolored adeno-associated viruses in the foreground and multicolored DNA strands in the background.
Quality Control for Cell and Gene Therapy
An orthogonal method to cell culture speeds up testing for AAV and lentivirus vectors.
Quality Control for Cell and Gene Therapy
Quality Control for Cell and Gene Therapy

An orthogonal method to cell culture speeds up testing for AAV and lentivirus vectors.

An orthogonal method to cell culture speeds up testing for AAV and lentivirus vectors.

gene therapy

An abstract illustration of a DNA helix and human lungs.
A New Delivery System Offers Hope for Cystic Fibrosis
Charlene Lancaster, PhD | May 1, 2024 | 4 min read
CRISPR-carrying lipid nanoparticles enabled researchers to correct a rare nonsense mutation in the lungs of a cystic fibrosis mouse model.
Different colored cartoon viruses entering holes in a cartoon of a human brain.
A Journey Into the Brain
Danielle Gerhard, PhD | Mar 22, 2024 | 10+ min read
With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.
Bringing Gene Therapy to the Brain
Bringing Gene Therapy to the Brain
The Scientist Staff | 1 min read
In this webinar, Douglas Marchuk and Viviana Gradinaru will discuss how scientists can overcome physiological barriers preventing gene therapies from reaching the brain. 
DNA molecule.
Finding DNA Tags in AAV Stacks
Mariella Bodemeier Loayza Careaga, PhD | Mar 7, 2024 | 8 min read
Ten years ago, scientists put DNA barcodes in AAV vectors, creating an approach that simplified, expedited, and streamlined AAV screening. 
Genotoxic Effects of Base and Prime Editing
Kamal Nahas, PhD | Jan 12, 2024 | 5 min read
A risk-benefit analysis of gene editing tools in stem cells revealed that base and prime editing carry vulnerabilities similar to those of CRISPR-Cas9, but at a reduced rate. 
Molecular illustration of CRISPR editing the DNA double helix
Prime Time Precision with CRISPR Technologies
The Scientist | 1 min read
Base editors and prime editors help researchers perform more precise in vivo and ex vivo translational research.
Abstract illustration depicting coronavirus research concept.
Curiosity and Compassion Fuel Rare Disease Research
The Scientist Staff | Jan 1, 2024 | 1 min read
Lauren Drouin shares how personal connections and scientific curiosities drive her work on gene therapy viral vectors. 
The Wonderfully Shrunken Cas13
Rachael Moeller Gorman | Nov 7, 2023 | 3 min read
Scientists removed unnecessary sections of the Cas13 enzyme, creating a mini-enzyme that works and fits with other CRISPR elements into a single gene therapy vector.
Illuminating Organs with Organoids
Illuminating Organs with Organoids
The Scientist | 1 min read
In this webinar, Benjamin Freedman talks about the importance of organoids in modeling organ function and disease.
Lipid nanoparticle
Which Gene Therapy Delivery Vector Will Emerge Victorious? 
Danielle Gerhard, PhD | Oct 2, 2023 | 2 min read
In the race to deliver successful gene therapies, frontrunner AAVs come head to head with underdog lipid nanoparticles. 
A man sitting at a desk in a white lab coat holds up a large model of a <em >Drosophila</em> fly. In the background is a window and a bookcase.
The Origins and Recent Promise of Nonsense Suppressor tRNAs
Ida Emilie Steinmark, PhD | Sep 8, 2023 | 4 min read
A discovery that goes back to the first studies of translation has become the topic of biotech buzz.
Equally-sized droplets of a nucleic acid sample.
Digital PCR: The Journey to Superior Data 
Bio-Rad Laboratories | 1 min read
With digital PCR (dPCR), researchers accurately and precisely quantitate nucleic acid samples.
A light gray mouse against an orange background listens to tiny headphones
Journey to the Center of the Ear
Niki Spahich, PhD | Aug 28, 2023 | 5 min read
An aqueduct connecting the brain to the ear may make gene therapy for hearing loss less invasive.
MRI images of brains from patients with epilepsy
Defending against Dravet
Aparna Nathan, PhD | Aug 21, 2023 | 3 min read
Gene therapy may be the first step toward curing a rare genetic epilepsy.
Building Bridges podcast logo
Building Bridges for Translational Research - A Special Podcast Series
The Scientist and Cytiva | 2 min read
Translational scientists discuss their experiences taking preclinical concepts to the market.
Patient with skin blisters being swabbed by gloved hand
Delivering Gene Therapies in Utero 
Natalia Mesa, PhD | Jul 18, 2023 | 3 min read
By delivering mRNA to the skin of mice in utero, researchers showed a proof-of-concept for shuttling gene therapies to skin cells before birth.
Learn How Researchers Make the Most of Viral Vectors for Gene Therapy
Viral Vector Platforms for Gene Therapy
The Scientist | Feb 9, 2023 | 1 min read
In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.
&nbsp;Learn about analyzing mRNA with direct sequence mapping&nbsp;
Solutions for mRNA Direct Sequence Mapping
Thermo Fisher Scientific | 1 min read
As mRNA therapeutics gain popularity, a novel solution for their development emerges.
2022 Top 10 Innovations&nbsp;
2022 Top 10 Innovations
The Scientist | Dec 12, 2022 | 10+ min read
This year’s crop of winning products features many with a clinical focus and others that represent significant advances in sequencing, single-cell analysis, and more.
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