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Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies
Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.
Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

disease & medicine, clinical trials

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.
Pelizaeus-Merzbacher disease neural stem cell transplantation
Neural Stem Cell Transplantation Crawls Toward the Clinic
Ashley P. Taylor | Oct 29, 2019 | 9 min read
Several early-stage clinical trials indicate that implanting patients with the cells is safe. But whether they can alleviate neurological problems remains to be seen.
UCB Cuts $2.1 Billion Deal to Acquire Ra Pharma
Catherine Offord | Oct 14, 2019 | 2 min read
The move will allow the Belgium-headquartered biopharmaceutical giant to boost its work on treatments for rare diseases.
Biogen, Eisai End Two Late-Stage Trials for Alzheimer’s Treatment
Emily Makowski | Sep 16, 2019 | 1 min read
The widely expected move comes months after the companies scrapped trials of another Alzheimer’s drug.
Recent Trials for Fragile X Syndrome Offer Hope
Randi Hagerman | Sep 1, 2019 | 10+ min read
Despite a solid understanding of the biological basis of fragile X syndrome, researchers have struggled to develop effective treatments.
Two Experimental Drugs Effective at Treating Ebola
Catherine Offord | Aug 13, 2019 | 2 min read
REGN-EB3 and mAb114 reduced mortality in a clinical trial carried out during the ongoing outbreak in Democratic Republic of Congo.
ebola vaccine uganda drc democratic republic of congo clinical trial
Uganda Launching New Experimental Ebola Vaccine Trial
Nicoletta Lanese | Aug 2, 2019 | 2 min read
Researchers aim to deploy the trial in neighboring Democratic Republic of Congo, which has been ravaged by Ebola for a year.
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Catherine Offord | Jul 26, 2019 | 1 min read
A trial is recruiting patients to test the gene-editing technology’s ability to treat an inherited form of blindness caused by a mutation in the CEP290 gene.
smart pills
Smart Pills Help Monitor Cancer Patients’ Therapy
Chia-Yi Hou | Jul 15, 2019 | 5 min read
Sensors encapsulated with oral chemotherapy drugs help patients and physicians keep track of treatments.
personalized vaccines
Infographic: Neoantigen Prediction for Personalized Vaccine Design
Jasreet Hundal and Elaine R. Mardis | Jul 15, 2019 | 3 min read
See how a computational pipeline uses next-generation sequencing data to identify genetic alterations that produce cancer-specific antigens.
CIRM California Institute for Regenerative Medicine funding running out not taking grant applications stem cell agency granting
Stem Cell Funding Agency CIRM Is Nearly Out of Funds
Chia-Yi Hou | Jul 8, 2019 | 3 min read
The California Institute for Regenerative Medicine has been funding research since it formed in 2004, giving out nearly $3 billion in grants to date.
fecal matter transplant FDA suspend halt clinical trials patient dies died antibiotic resistance drug resistant
FDA Suspends Clinical Trials Involving Fecal Transplants
Chia-Yi Hou | Jun 14, 2019 | 1 min read
The agency issued a safety warning after two patients contracted antibiotic-resistant infections and one of the patients died.
natural killer cells nk cell therapy product immunotherapy clinical trial
Clinical Trial Underway for a Natural Killer Cell Therapy
Chia-Yi Hou | May 7, 2019 | 3 min read
The cells, derived from induced pluripotent stem cells, are in testing as an immunotherapy for cancer patients with solid tumors.
Donor-Derived iPS Cells Show Promise for Treating Eye Disease
Jef Akst | Apr 30, 2019 | 2 min read
Age-related macular degeneration patients who received injections of retinal cells derived from donors’ induced pluripotent stem cells have maintained their level of eyesight for a year.
peanut allergy anaphylaxis oit oral immunotherapy
Peanut Allergy Immunotherapy Increases Anaphylaxis Risk: Study
Kerry Grens | Apr 26, 2019 | 2 min read
Although the oral treatment seems to work, an analysis of the results from 12 clinical trials finds kids who got an immunotherapy have a greater rate of serious reactions.
april 2019 crossword
Ten-Minute Sabbatical
The Scientist | Apr 1, 2019 | 2 min read
Take a break from the bench to puzzle and peruse.
Esketamine, a Treatment for Depression, Receives FDA Approval
Carolyn Wilke | Mar 6, 2019 | 2 min read
The nasal spray to ease intractable depression appears on the US market after decades without novel antidepressant treatments.
Japan Approves iPS Cell Therapy Trial for Spinal Cord Injury
Catherine Offord | Feb 18, 2019 | 1 min read
The treatment will be tested in a handful of patients who suffered nerve damage in sports or traffic accidents.
Ebola Clinical Trial to Begin Amid Outbreak in DRC
Ashley P. Taylor | Nov 12, 2018 | 2 min read
The study will examine the efficacy of four drugs—an antiviral and three monoclonal antibodies—that are already being used to treat patients in Democratic Republic of Congo.
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