Nexiguran ziclumeran (nex-z), an investigational CRISPR-Cas9 gene therapy for transthyretin amyloid cardiomyopathy (ATTR-CM), continues to demonstrate long-term efficacy in early-phase research, even as its phase III trials remain on hold…
Editor's Note: Patient Worthy is proud to share this article from our friends at the TESS Research Foundation. To see the article in its original format, please click here. TL;DR: CRISPR…
When baby KJ was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a rare metabolic disorder that can trigger life-threatening ammonia buildup, his family faced a narrow path: strict dietary…
According to an article in Inside Precision Medicine Researchers have developed a new, easy-to-use test called SHINE-TB that can quickly diagnose tuberculosis (TB) using saliva samples. This test is based…
Researchers at the UK Dementia Institute in London (UK DRI) have identified a common denominator for various gene mutations that cause amyotrophic lateral sclerosis (ALS). A new CRISPR study shed…
Researchers discovered a version of the Cas protein that fits into adeno-associated viruses (AAVs). This virus can be engineered to deliver targeted therapy to cells and is a common approach…
Recently there has been an increasing amount of research attempting to control CRISPR gene-editing systems. These systems are found in many types of bacteria as a defense against viruses. The…
CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…
Leukemia is cancer that begins in the bone marrow and involves abnormal white blood cells (WCB) from lymphatic tissues and bone marrow. Scientists have new theories as to why certain…
Business Wire published an August 10th news release announcing that Cure Rare Disease, a non-profit based in Boston, Massachusetts received FDA approval to administer CRD-TMH-001 (CRD), its first-ever therapeutic.…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR,…
In a December 13 press release listed on the company's website, genome editing company Intellia Therapeutics, Inc. ("Intellia") shared that the first patient had been dosed in a Phase…
CRISPR Cas9 gene editing is still looked upon as a new phenomenon in medicine. Yet it appears in the news almost every week. This week Neurology Today shines a light…
CRISPR is a gene-editing technique that has revolutionized the medical world with its approach to making precise changes in DNA. CRISPR found its match in 35-year-old Victoria Gray of…
CRISPR (clustered regularly interspaced short palindromic repeats) was discovered over a decade ago by Emmanuelle Charpentier and Jennifer Doudna, two scientists who recently shared the Nobel Prize. Scientists using…
According to a recent news item that appeared on The Denver Channel, we are now in the age of genetic research that could have an impact on the lives of…
Over ten years ago two Nobel-Prize-winning biologists, Emmanuelle Charpentier and Jennifer Doudna, discovered a technology called CRISPR (clustered regularly interspaced short palindromic repeats). Bacteria contain genetic sequences repeated throughout…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
The Hindustan Times recently published an article that again puts CRISPR at the forefront of medical news. The newest CRISPR tool was designed to identify Sars-CoV-2 and prevent the transmission…
Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…
CRISPR is a gene-editing technology which holds the potential to treat a variety of genetic diseases and improve patient outcomes. Overall, CRISPR works by creating precise cuts and edits within…
Medscape reported that when Victoria Gray was three months old, her family was told to keep her close: her prognosis wasn't looking good. She had been rushed to the ER…
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
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