Participant’s Diagnosis Halts Gene Therapy Clinical Trial
Participant’s Diagnosis Halts Gene Therapy Clinical Trial
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.
Participant’s Diagnosis Halts Gene Therapy Clinical Trial
Participant’s Diagnosis Halts Gene Therapy Clinical Trial

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.

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.

gene therapy
Gene Therapy Continues to Benefit Kids with Immunodeficiency
Gene Therapy Continues to Benefit Kids with Immunodeficiency
Jef Akst | May 12, 2021
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.
Laurence “Larry” Kedes, Molecular Geneticist, Dies at 83
Laurence “Larry” Kedes, Molecular Geneticist, Dies at 83
Amanda Heidt | Apr 26, 2021
In addition to isolating the first protein-coding gene from a eukaryote, Kedes furthered scientists’ understanding of actin genes and also laid the foundations for modern DNA databases such as GenBank.
Gene Therapy for Sickle Cell Not Linked to Cancer, Bluebird Finds
Gene Therapy for Sickle Cell Not Linked to Cancer, Bluebird Finds
Lisa Winter | Mar 11, 2021
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.
Gene Therapy in One Eye Improves Vision in Both Eyes
Gene Therapy in One Eye Improves Vision in Both Eyes
Abby Olena | Dec 11, 2020
It’s not clear why the patients with Leber hereditary optic neuropathy, a mitochondrial disorder that causes blindness, also experienced the modest benefits in their untreated eye.
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Emma Yasinski | Sep 25, 2020
Adeno-associated viral vectors can deliver gene therapies, but AAV-neutralizing antibodies might prevent the medicines from working.
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.
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.