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Learn How Researchers Make the Most of Viral Vectors for Gene Therapy
Viral Vector Platforms for Gene Therapy
In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.
Viral Vector Platforms for Gene Therapy
Viral Vector Platforms for Gene Therapy

In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.

In both the laboratory and clinic, scientists harness viral genetic transfer capabilities to develop gene therapies that modulate cellular function.

genetics & genomics, drug development

Red blood vessels that decrease in diameter as they radiate outward are pictured on a pink and white surface
Antisense Oligonucleotides Cross Rodents’ Blood-Brain Barrier
Abby Olena, PhD | Aug 18, 2021 | 3 min read
RNA-DNA complexes that were modified with cholesterol made it into the brains of rats and mice, where they knocked down target genes.
An artistic rendering of blue neurons against a white background
Participant’s Diagnosis Halts Gene Therapy Clinical Trial
Amanda Heidt | Aug 12, 2021 | 3 min read
The FDA pauses the research program on a lentivirus-based treatment for a rare neurological condition after a patient developed a bone marrow disorder that could presage leukemia.
An illustration of a DNA double helix in gold with texture
Gene Therapy Continues to Benefit Kids with Immunodeficiency
Jef Akst | May 12, 2021 | 2 min read
Four dozen children with severe combined immunodeficiency (SCID) who received a corrective gene carried by a virus have working immune systems two to three years later, according to three independent clinical trials.
Gene Therapy for Sickle Cell Not Linked to Cancer, Bluebird Finds
Lisa Winter | Mar 11, 2021 | 2 min read
Clinical trials were halted after the treatment’s vector that ferries in the healthy genetic sequence was identified in the genome of a patient’s cancer cells.
Vaccines Versus the Mutants
Anthony King | Feb 8, 2021 | 7 min read
Facing new variants of SARS-CoV-2, some vaccines may offer more robust protection or be more easily redesigned to target them.
Kathryn Anderson, forward genetics, genetics & genomics, model organisms, Toll, hedgehog, embryogenesis, developmental biology, cell differentiation, cilia,
Developmental Biologist Kathryn Anderson Dies at 68
Amanda Heidt | Jan 6, 2021 | 4 min read
The Sloan Kettering researcher used mutagenic screening to probe genes and molecular pathways, including Toll and Hedgehog, essential to development in fruit flies and mice.
Gene Therapy Finds a Fertile Home in Ohio
Shawna Williams | Jan 13, 2020 | 8 min read
The midwestern state has quietly laid the groundwork for a biotech hub.
Transposons Identified as Likely Cause of Undiagnosed Diseases
Jef Akst | Jan 13, 2020 | 4 min read
A tool for identifying jumping gene insertions in DNA sequencing data turns up possible explanations for four patients’ rare developmental disorders.
3d printed rabbit dna data storage
Object Engineered to Carry a DNA Code for its Own Replication
Kerry Grens | Dec 9, 2019 | 2 min read
A 3-D printed rabbit is made from a blueprint stored in DNA, which itself is stored in a printed rabbit.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019 | 2 min read
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
geneplaza how gay am i genetic gwas homosexual heterosexual same-sex sexual behavior
Scientists Seek to Kill Genetic Test for Same-Sex Attraction
Diana Kwon | Oct 17, 2019 | 4 min read
Direct-to-consumer “How Gay Are You?” app sold by GenePlaza has been criticized for being misleading and irresponsible. 
Recent Trials for Fragile X Syndrome Offer Hope
Randi Hagerman | Sep 1, 2019 | 10+ min read
Despite a solid understanding of the biological basis of fragile X syndrome, researchers have struggled to develop effective treatments.
a DNA helix made of fruits and vegetables
Personalized Nutrition Companies’ Claims Overhyped: Scientists
Shawna Williams | Aug 21, 2019 | 8 min read
Services promising scientifically tailored supplements or advice abound. Experts say their claims are largely unfounded, although some solid evidence is beginning to emerge.
nancy davidson fred hutch epigenetics breast cancer
Q&A: Epigenetic Therapies for Breast Cancer
Katarina Zimmer | Apr 4, 2019 | 4 min read
Breast cancer researcher and oncologist Nancy Davidson discusses what we’ve learned from the first wave of epigenetic trials for breast cancer, and what challenges lie ahead before such therapies reach the clinic.
Emily Derbyshire scientist to watch
Emily Derbyshire Looks for Malaria’s Vulnerabilities
Shawna Williams | Mar 1, 2019 | 3 min read
The Duke University professor studies the parasite to find a way to thwart infection before it takes hold.
Opinion: The Central Dogma of Mitochondrial Genetics Needs Rewriting
John D. Loike | Dec 12, 2018 | 3 min read
The recent discovery of 17 people who have inherited maternal and paternal lines of mitochondrial DNA has major ramifications for medical and ancestry research.
Ancient Andeans Had Novel Genetic Advantages to Adapt to Altitude
Ashley Yeager | Oct 19, 2018 | 3 min read
Unlike other populations living at high altitude, Andeans didn’t rely on hypoxia-related genes.
Scientists Play Favorites with Studying Human Genes. Here’s Why.
Sukanya Charuchandra | Sep 19, 2018 | 2 min read
Despite the Human Genome Project having heralded the exploration of previously unknown human genes, the focus of genetic studies remains narrow.
The NIH Loosens Grip on Gene Therapy Trials
Kerry Grens | Aug 16, 2018 | 2 min read
The agency proposes ceding its scrutiny of these studies to the FDA.
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