Building with sign reading “Novartis”
Two Children Die After Receiving Novartis Gene Therapy
No deaths had previously been associated with the muscle-wasting treatment Zolgensma.
Two Children Die After Receiving Novartis Gene Therapy
Two Children Die After Receiving Novartis Gene Therapy

No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

No deaths had previously been associated with the muscle-wasting treatment Zolgensma.

gene therapy
A white mouse huddles with some of her nine-day-old pups.
In Vivo Gene Therapy Cures Infertility in Mice
Dan Robitzski | May 2, 2022
Mice rendered infertile through ovary cell–targeting mutations gave birth to seemingly normal offspring through natural mating after a virus-based gene therapy was injected into their ovaries.
An orange CRISPR Cas 9 enzyme cutting DNA
CRISPR-Based Treatment Successfully Lowers Toxic Protein Levels
Natalia Mesa | Mar 2, 2022
A first-of-its-kind gene therapy dramatically reduced misfolded protein levels in some clinical trial participants for up to six months and reduced levels in all participants for up to a year.
Learn How Cutting-Edge Science is Breaking Through Industry Barriers
Focus on Innovation: Scientific Advances Driving Biopharma Market Trends
The Scientist Creative Services Team and Tecan
Explore the future of biopharma research and development.
DNA
In Editing RNA, Researchers See Endless Possibilities
Christie Wilcox | Dec 1, 2021
RNA editing has been in DNA editing’s shadow for nearly a decade, but recent investments in the technology could bring it into the limelight.
An artistic rendering of blue neurons against a white background
Participant’s Diagnosis Halts Gene Therapy Clinical Trial
Amanda Heidt | Aug 12, 2021
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
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
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.
3D virus cells attacking a DNA strand
HIV DNA Circularizes to Bypass CRISPR-Based Treatments
Nele Haelterman, PhD
CRISPR-mediated removal of HIV can create small, infectious DNA molecules.
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
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.
The Past, Present, and Future of Cell and Gene Therapy
The Past, Present, and Future of Cell and Gene Therapy
The Scientist Creative Services Team and Bio-Rad Laboratories
Embark on the journey of cell and gene therapy—from its conception and development, to its present state, and into its future.
aav adeno-associated virus vector gene therapy antibody hemophilia
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
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.
800x560-thermo-october
A Scalable Process to Efficiently Manufacture Adeno-Associated Viral Vectors
The Scientist Creative Services Team in collaboration with Thermo Fisher Scientific
An optimized AAV production system allows researchers to consistently generate high virus titers.
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
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
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.
Harnessing Stem Cells to Model Neurological Disorders
The Scientist Creative Services Team
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.