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Two hands holding tweezers and dissecting single points in a DNA sequence.
From CRISPR to Prime Editing: The Evolution of the Genome Editing Revolution
Even as CRISPR-based tools become a lab staple, scientists strive to tackle the associated technological challenges to improve their efficacy and safety.   
From CRISPR to Prime Editing: The Evolution of the Genome Editing Revolution
From CRISPR to Prime Editing: The Evolution of the Genome Editing Revolution

Even as CRISPR-based tools become a lab staple, scientists strive to tackle the associated technological challenges to improve their efficacy and safety.   

Even as CRISPR-based tools become a lab staple, scientists strive to tackle the associated technological challenges to improve their efficacy and safety.   

gene therapy

Abstract, colorful illustration of the human brain
A Gene Therapy to Treat the FOXG1 Brain Disorder 
Niki Spahich, PhD | Sep 12, 2024 | 4 min read
By postnatally providing a transcription factor important for brain development, researchers fixed abnormalities generated in utero in mice.
An artist’s representation of the human respiratory system with pink airways on a blue and black background.
A Gene Editing System Corrects Cystic Fibrosis Mutation
RJ Mackenzie | Sep 4, 2024 | 4 min read
An optimized version of prime editing technology raises the possibility of a one-time treatment for cystic fibrosis.
An illustration of an adeno-associated virus.
Streamlining Viral Gene Therapy Development
The Scientist and Charles River Laboratories, Inc. | 3 min read
Off-the-shelf and custom plasmid DNA and viral vector products help scientists take their gene therapy research to the next level.
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
The Scientist Staff | Jul 1, 2024 | 2 min read
An orthogonal method to cell culture speeds up testing for AAV and lentivirus vectors.
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.
Pharmaceutical manufacturing facility
GMP-Grade Streamlines Cell and Gene Therapy Manufacturing
ACROBiosystems | 1 min read
Standardized quality materials and reagents help scientists improve cell and gene therapy production.
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.
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. 
iStock
Catch Me If You Can: Sequencing Screens for Rare Disease Genes
The Scientist | 1 min read
As a trailblazer in rare disease research and treatment, Wendy Chung captures the big picture of rare disease genetics with the help of next generation sequencing.
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. 
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. 
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. 
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.
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. 
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.
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.
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.
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.
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.
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