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Different colored cartoon viruses entering holes in a cartoon of a human brain.
A Journey Into the Brain
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.
A Journey Into the Brain
A Journey Into the Brain

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.

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.

gene therapy, neuroscience

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.
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.
Harnessing Stem Cells to Model Neurological Disorders
The Scientist | Apr 15, 2021 | 2 min read
CRISPR Technologies for the New Era of Cell and Gene Therapy
The Scientist | Nov 25, 2020 | 1 min read
Experts will discuss how they use new CRISPR technologies to advance their cell and gene therapy research.
Immune-Activating Gene Therapy for Glioblastoma
Ruth Williams | Aug 14, 2019 | 3 min read
The results of an early trial in 31 brain cancer patients finds immune activity boosted in the tumor, and possibly longer survival.
Reprogrammed Müller Glia Restore Vision in Mice
Ashley Yeager | Aug 15, 2018 | 4 min read
A double gene-transfer therapy transformed the non-neuronal cells into rod photoreceptors in the retinas of animal models of congenital blindness.
A Noninvasive Way to Control Individual Brain Regions
Catherine Offord | Jul 13, 2018 | 2 min read
Researchers use a combination of ultrasound waves, genetic engineering, and synthetic drugs to switch specific neurons on and off in mice.
Study Pinpoints Potential “Master Regulator” of Age-Related Cognitive Decline
Shawna Williams | Dec 18, 2017 | 4 min read
Upping a gene’s expression in rat brains made them better learners and normalized the activity of hundreds of other genes to resemble the brains of younger animals.
Hearing Channel Components Mapped
Kerry Grens | Sep 4, 2015 | 2 min read
Localization of two proteins important for inner ear hair cell function suggests they are part of the elusive mechanotransduction channel. 
Gene Therapy Fixes Mouse Hearing
Kerry Grens | Jul 9, 2015 | 1 min read
Expressing a gene for a component of the inner ear’s hair cells treated a form of genetic deafness.
Opinion: The Payoff of Big Science
K. John Morrow Jr. | Jun 3, 2013 | 4 min read
Was the Human Genome Project the key to a gold mine?
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