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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.

gene therapy, genetics & genomics

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.
obituary, obituaries, microbiology, molecular genetics, biochemistry, bioinformatics, Stanford University, University of Southern California, cell & molecular biology
Laurence “Larry” Kedes, Molecular Geneticist, Dies at 83
Amanda Heidt | Apr 26, 2021 | 4 min read
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
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.
Gene Therapy in One Eye Improves Vision in Both Eyes
Abby Olena, PhD | Dec 11, 2020 | 4 min read
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.
aav adeno-associated virus vector gene therapy antibody hemophilia
Thwarting AAV-Neutralizing Antibodies Could Improve Gene Therapy
Emma Yasinski | Sep 25, 2020 | 5 min read
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
Lisa Winter | Sep 4, 2020 | 2 min read
The International Commission on the Clinical Use of Human Germline Genome Editing claims the technology is still too risky for therapeutic use.
gene, CRISPR, CRISPR-Cas9, gene editing, human embryo, chromosome, mutation, deletion
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
Amanda Heidt | Jun 26, 2020 | 3 min read
Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.
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.
a baby's hand cradled by an adult hand
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020 | 2 min read
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
sma spinal muscular atrophy zolgensma in utero fetal gene therapy smn1
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez, PhD | Dec 11, 2019 | 4 min read
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
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.
gene therapy approval used manipulated data
Some Data Backing Novartis Gene-Therapy Approval Manipulated: FDA
Nicoletta Lanese | Aug 7, 2019 | 2 min read
The agency is now evaluating the implications of the corrupted data behind Zolgensma and whether to “take action” against the pharmaceutical company.
FDA Approves Gene Therapy for Spinal Muscular Atrophy
Ashley Yeager | May 27, 2019 | 3 min read
At $2 million for a single dose, Novartis’s Zolgensma is the most expensive medicine to date, but still less expensive over a lifetime than another approved drug for the rare genetic disease.
gene therapy mouse mice model retinal disease retinitis pigmentosa
Image of the Day: Cone Preservation
Chia-Yi Hou | Apr 30, 2019 | 1 min read
Researchers treat mice with retinitis pigmentosa using a gene therapy that reduces cone loss.
a drop of blood for a newborn's heel is placed on a piece of paper
Gene Therapy Effective for Severe Combined Immunodeficiency
Shawna Williams | Apr 17, 2019 | 4 min read
Researchers report they’ve found a way to restore immune function in infants with one form of “bubble boy disease.”
Preliminary Results Point to Success of In Vivo Gene Editing
Carolyn Wilke | Feb 12, 2019 | 2 min read
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
Early Results of First In Vivo Gene-Editing Trial Encouraging
Kerry Grens | Sep 5, 2018 | 2 min read
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.
CRISPR Treatment for Duchenne Muscular Dystrophy Helps Dogs
Catherine Offord | Aug 31, 2018 | 2 min read
Researchers boosted levels of the dystrophin protein to nearly normal levels in a canine model of the disease.
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