Gene Therapy Continues to Benefit Kids with Immunodeficiency
Gene Therapy Continues to Benefit Kids with Immunodeficiency
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.
Gene Therapy Continues to Benefit Kids with Immunodeficiency
Gene Therapy Continues to Benefit Kids with Immunodeficiency

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.

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.

gene therapy
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.
Harnessing Stem Cells to Model Neurological Disorders
Harnessing Stem Cells to Model Neurological Disorders
The Scientist Creative Services Team | Apr 15, 2021
Infographic: The Value of Scaling: PSC Suspension Cultures
Infographic: The Value of Scaling: PSC Suspension Cultures
Thermo Fisher Scientific | Mar 17, 2021
As research and therapeutic applications for pluripotent stem cells (PSCs) grow, so does the need to reliably and cost-effectively generate large numbers of high-quality cells.
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.
Optimizing Adeno-Associated Virus (AAV) Manufacturing
Optimizing Adeno-Associated Virus (AAV) Manufacturing
The Scientist Creative Services Team in collaboration with OXGENE | Feb 16, 2021
Scientists wield nature’s power to optimize adeno-associated virus (AAV) production and maximize gene therapy safety.
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.
<em>The Scientist</em>&#39;s LabTalk - Episode 4
The Scientist's LabTalk - Episode 4
The Scientist Creative Services Team | Dec 11, 2020
The Past, Present, and Future of Gene Therapy: How to Scale-up Successfully
CRISPR Technologies for the New Era of Cell and Gene Therapy
CRISPR Technologies for the New Era of Cell and Gene Therapy
The Scientist Creative Services Team | Nov 25, 2020
Experts will discuss how they use new CRISPR technologies to advance their cell and gene therapy research.
Gene Therapy Workflow from Production to Quality Control
Gene Therapy Workflow from Production to Quality Control
The Scientist Creative Services Team | Oct 14, 2020
Learn about some instruments used in gene therapy production.
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.
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.