Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy

Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.

Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.

gene therapy
Panel Lays Out Guidelines for CRISPR-Edited Human Embryos
Panel Lays Out Guidelines for CRISPR-Edited Human Embryos
Lisa Winter | Sep 4, 2020
The International Commission on the Clinical Use of Human Germline Genome Editing claims the technology is still too risky for therapeutic use.
The Scientist Speaks Podcast - Episode 8
The Scientist Speaks Podcast - Episode 8
The Scientist Creative Services Team | Aug 26, 2020
Experimental Cures for Fragile Patients: Prenatal Stem Cell and Gene Therapies
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
Amanda Heidt | Jun 26, 2020
Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Abby Olena | Apr 10, 2020
Researchers slowed disease progression in the mice by injecting two different viral vectors, each containing one part of the DNA encoding the Cas9 protein, to edit the causative gene.
First Patient Receives In Vivo CRISPR Editing
First Patient Receives In Vivo CRISPR Editing
Jef Akst | Mar 4, 2020
Doctors in Oregon delivered the gene editing machinery behind the retina in hopes of treating an inherited form of blindness, according to the companies that developed the therapy.
Gene Therapy Finds a Fertile Home in Ohio
Gene Therapy Finds a Fertile Home in Ohio
Shawna Williams | Jan 13, 2020
The midwestern state has quietly laid the groundwork for a biotech hub.
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy
Jef Akst | Jan 6, 2020
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez | Dec 11, 2019
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.