What Is CRISPR?
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…
Continue Reading
What Is CRISPR?
World’s First Personalized Gene-Editing Therapy Offers Hope to Infant with Rare Disease
In a groundbreaking medical achievement, physicians have administered the world’s first personalized gene-editing therapy to an infant suffering from a rare and fatal genetic disorder. The innovative treatment, described at…
Continue Reading
World’s First Personalized Gene-Editing Therapy Offers Hope to Infant with Rare Disease
source: shutterstock.com
DNA Base and Prime Editing Could Correct Hemophilia A Gene Mutations
Genetic disorders are caused by genetic mutations (changes) in your gene. Over the past decade especially, researchers increasingly explore genome editing options to fix or address genetic diseases at the…
Continue Reading
DNA Base and Prime Editing Could Correct Hemophilia A Gene Mutations
source: shutterstock.com
Doctors Transplant Genetically Edited Pig Kidney Into 62-Year-Old Man with End-Stage Kidney Disease
Over 100,000 individuals living in the U.S. are on a waiting list for an organ transplant. An average of 17 people die each day while waiting for a donor organ.…
Continue Reading
Doctors Transplant Genetically Edited Pig Kidney Into 62-Year-Old Man with End-Stage Kidney Disease
source: shutterstock.com
U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell
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…
Continue Reading
U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell
Photo by Hush Naidoo Jade Photography on Unsplash
VERVE-101 Shows Promise in Treating HeFH, Phase 1b Study Results Show
Heterozygous familial hypercholesterolemia (HeFH) can be difficult to control on available standard-of-care cholesterol-lowering medications. Therapeutic interventions are needed to reduce high LDL cholesterol levels and improve health and quality-of-life for…
Continue Reading
VERVE-101 Shows Promise in Treating HeFH, Phase 1b Study Results Show
source: shutterstock.com
Nanoparticles Delivering mRNA Gene Editing Solutions to the Lungs Can Combat Genetic Diseases
Treatment advances are occurring daily in the field of rare disease. As researchers hone their strategies and tools, new technologies are emerging with the potential to significantly impact patients…
Continue Reading
Nanoparticles Delivering mRNA Gene Editing Solutions to the Lungs Can Combat Genetic Diseases
source: pixabay.com
Gene Editing in Ticks May Help Prevent Tick-Borne Diseases
According to a recent article from MedicineNet, scientists are editing the genomes of ticks to help prevent tick-borne diseases such as Lyme disease. Lyme Disease Lyme disease is a vector-borne…
Continue Reading
Gene Editing in Ticks May Help Prevent Tick-Borne Diseases
https://pixabay.com/photos/hospital-infusion-hand-association-834157/
First Patient Dosed in NTLA-2002 Trial for HAE
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…
Continue Reading
First Patient Dosed in NTLA-2002 Trial for HAE
CRISPR/Cas9 Technology May Be the First Treatment to Cure ATTR Amyloidosis
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…
Continue Reading
CRISPR/Cas9 Technology May Be the First Treatment to Cure ATTR Amyloidosis
CRISPR’s New Technique and Transthyretin Amyloidosis
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…
Continue Reading
CRISPR’s New Technique and Transthyretin Amyloidosis
source: pixabay.com
Clinical Trial to Evaluate HMI-103 for PKU
Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…
Continue Reading
Clinical Trial to Evaluate HMI-103 for PKU
source: pixabay.com
CRISPR: It Has Only Just Begun
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…
Continue Reading
CRISPR: It Has Only Just Begun
PublicDomainPictures / Pixabay
Gene Editing and the Future of FSHD
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…
Continue Reading
Gene Editing and the Future of FSHD
CRISPR-Cas9 Has Achieved Worldwide Acceptance
Crispr is no longer a buzzword. According to a recent article in The Guardian, it has become the ‘molecular scissors’ that will enable scientists to rewrite our genes or as often said,…
Continue Reading
CRISPR-Cas9 Has Achieved Worldwide Acceptance
source: pixabay.com
Could Gene Editing Prevent Alzheimer’s?
What if there was a way to genetically prevent the development of certain conditions, such as Alzheimer's disease? According to Medical XPress, this might soon be an option on the…
Continue Reading
Could Gene Editing Prevent Alzheimer’s?
fernandozhiminaicela / Pixabay
RNA Editing Offers Benefits Over CRISPR
In 2012, a new medical technology emerged: CRISPR-Cas9, which offered the opportunity for gene editing. LabBiotech explains that gene editing could benefit patients with genetic disorders by adding, deleting, or…
Continue Reading
RNA Editing Offers Benefits Over CRISPR
Is Angelman Syndrome Gene Deletion Therapy On the Horizon?
According to Angelman Syndrome News, researchers used rat models of Angelman syndrome to test gene editing as a potential therapy. They found that CRISPR technology deleted the defective UBE3A gene. However, rats…
Continue Reading
Is Angelman Syndrome Gene Deletion Therapy On the Horizon?
Sourced from NIH Image Gallery (Public Domain). Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH
CRISPR and COVID-19: The Impact on Biotech
Saying that COVID-19 has impacted the world might just be an understatement. In fact, COVID-19 changed the way we interact with each other, businesses, and the world around us. But how…
Continue Reading
CRISPR and COVID-19: The Impact on Biotech
mcmurryjulie / Pixabay
CRISPR’s First Patient Share Her Story
As originally reported in Medical Xpress, CRISPR has been a hot conversation starter in recent years. It has been framed as gene editing made easy: cheaper and simpler than previous…
Continue Reading
CRISPR’s First Patient Share Her Story
ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects
We've been hearing A LOT about so-called 'gene editing' and 'CRISPR' lately -- and now there's new developments on that front. Scientists have developed a new gene-editing technology called 'prime…
Continue Reading
ICYMI: New Form of Gene Editing Could Correct 89% of Genetic Defects
PublicDomainPictures / Pixabay
Biomedical Innovation Law Expert Presents Ethical Difficulties Posed by Gene Editing
According to a publication from Charcot Marie Tooth News, biomedical law expert Timo Minssen recently gave a presentation at the New York Genome Center where he discussed the potential ethical…
Continue Reading
Biomedical Innovation Law Expert Presents Ethical Difficulties Posed by Gene Editing
source: pixabay.com
Plan Proposed to Use CRISPR to Edit Gene Carrying HIV by Russian Biologist
As reported in RadioFreeEurope, Russian biologist Denis Rebrikov is currently challenging the norm of caution regarding gene-editing in the science world, with efforts to gain the go-ahead from Russia to…
Continue Reading
Plan Proposed to Use CRISPR to Edit Gene Carrying HIV by Russian Biologist
Could Gene Editing Pigs Be the Future of Treating Rare Diseases?
The Atlantic featured this incredible story about the possibilities of using genetically altered piglets as avatars for children with genetic diseases. Furthermore, the article delves into the personal story of one…
Continue Reading
Could Gene Editing Pigs Be the Future of Treating Rare Diseases?
mohamed_hassan / Pixabay
Watch: HBO’s John Oliver Deep Dives into Gene Editing/CRISPR
For any John Oliver fans out there, you know his thing is deep-diving into topics of importance - from beauty pageants to international elections to failing American infrastructure - just…
Continue Reading
Watch: HBO’s John Oliver Deep Dives into Gene Editing/CRISPR
