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tag gene therapy immunology disease medicine

DNA molecule.
Finding DNA Tags in AAV Stacks
Mariella Bodemeier Loayza Careaga, PhD | Mar 7, 2024 | 8 min read
Ten years ago, scientists put DNA barcodes in AAV vectors, creating an approach that simplified, expedited, and streamlined AAV screening. 
Blue T cell with other blurred T cells in the background
Woman Seemingly Cured of HIV After Umbilical Cord Transplant
Natalia Mesa, PhD | Feb 16, 2022 | 3 min read
Umbilical cord blood may be a good alternative to bone marrow transplants for treating HIV in patients with HIV and cancer.
Photo of Rosa Bacchetta, associate professor of pediatrics at Stanford University
The Future of Gene Therapy for a Rare Pediatric Autoimmune Disease
Niki Spahich, PhD | Jul 10, 2023 | 3 min read
By editing a mutated immune regulatory gene in patient cells, Rosa Bacchetta brings hope to those suffering from IPEX syndrome.
A needle drawing up fluid from an unlabeled vial.
Cancer Vaccination as a Promising New Treatment Against Tumors
Shelby Bradford, PhD | Mar 15, 2024 | 10+ min read
Vaccination has beaten back infections for more than a century. Now, it may be the next big step in battling cancer.
A bat flying in a dark cave
Turning on the Bat Signal
Hannah Thomasy, PhD | Mar 15, 2024 | 10+ min read
Scientists around the world investigate how bat immune systems cope with viral attacks and how this information could be used to keep humans safe.
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.
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.”
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.
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.
The Breakthrough Prize ?Trophy
2024 Breakthrough Prizes in Life Sciences
Danielle Gerhard, PhD | Sep 14, 2023 | 10 min read
This year’s Breakthrough Prizes honor advances in CAR T cancer therapies, cystic fibrosis, and Parkinson’s disease.

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