Breaking News
May 25, 2018 - TNFi Exposure In Utero Does Not Up Serious Infection Risk
May 25, 2018 - Organization of cells in the inner ear enables the sense and sensitivity of hearing
May 25, 2018 - Yoga May Be Right Move Against Urinary Incontinence
May 25, 2018 - New cytokine network can repair tissue damage in the intestine, study finds
May 25, 2018 - Lyme disease researcher dispels misconceptions about ticks and provides prevention tips
May 25, 2018 - Penn researchers find link between social media usage and underage drinking
May 25, 2018 - Unique nanotechnology method to simplify skin disease diagnosis
May 25, 2018 - Study reveals new protective mechanism for tumor cells in breast cancer
May 25, 2018 - FRAME Alternatives Laboratory chosen for major European liver research collaboration
May 25, 2018 - Study shows yogurt may dampen chronic inflammation linked to multiple diseases
May 25, 2018 - Invasive cancers that are born to be bad show detectable differences from harmless tumors
May 25, 2018 - Study identifies new mechanism involved in development of Lou Gehrig’s disease
May 25, 2018 - UAB professor receives award for malaria prevention study in pregnant women in Cameroon
May 25, 2018 - Study provides blueprint of how fruit flies can be used to screen potentially pathogenic human genes
May 25, 2018 - New drug-delivering nanoparticle could offer better way to treat brain tumors
May 25, 2018 - Kessler Foundation scientists compare two tests for assessing learning in individuals with MS
May 25, 2018 - Stroke Symptoms and Diagnosis (Beyond the Basics)
May 25, 2018 - Protein goes against the family to prevent cancer
May 25, 2018 - Drugmakers blamed for blocking generics have milked prices and cost U.S. billions
May 25, 2018 - Speakers announced for National Medicines Symposium 2018
May 25, 2018 - GSK Receives FDA Approval of Arnuity Ellipta for Asthma in Children From 5 Years of Age
May 25, 2018 - Pfizer settles kickback case related to copay assistance for $24m
May 25, 2018 - Nuclear pore functions are essential for T cell survival
May 25, 2018 - Study defines molecular basis to explain connection between mother’s nutrition and infant growth
May 24, 2018 - IHI hosts representatives to develop a national action plan for patient safety
May 24, 2018 - Zika detection breakthrough by University of Queensland
May 24, 2018 - FDA Alert: 95% Ethyl Alcohol Product by Ethanol Extraction: Recall
May 24, 2018 - New method allows scientists to study how HIV persists
May 24, 2018 - Study reveals rate of vertebral and non-vertebral fractures in children with leukemia
May 24, 2018 - Whey protein supplementation and physical activity aid women in improving body composition
May 24, 2018 - Seniors’ air pollution exposure linked to hospitalization for ARDS
May 24, 2018 - Home-based telehealth therapy program effective for stroke rehabilitation, shows study
May 24, 2018 - Addressing Parents’ HPV Vaccine Hesitancy Ups Vaccination Rates
May 24, 2018 - Opioid addiction treatment drug helps suppress HIV in former prisoners
May 24, 2018 - FDA warns against using teething remedies for babies
May 24, 2018 - Healthy lifestyle counseling program linked to reduced risk of developing cancers
May 24, 2018 - CU research sheds light on liver disease caused by intravenous nutrition
May 24, 2018 - Skin cream containing rapamycin reduces TSC-related facial tumors
May 24, 2018 - Suicide rates twice as high among black children finds new study
May 24, 2018 - Researchers find new method to treat severe asthma
May 24, 2018 - Scientists report new strategy for fighting bacteria
May 24, 2018 - South Asians living in the United States more likely to die of heart disease and stroke
May 24, 2018 - Health Tip: Why Get a Biopsy
May 24, 2018 - Metabolic Syndrome Prevalence by Race/Ethnicity and Sex in the United States, National Health and Nutrition Examination Survey, 1988–2012
May 24, 2018 - Motivation to move may start with being mindful
May 24, 2018 - Advanced genetics study of TB bacteria uncovers virulent ‘Beijing lineage’ strain among young adults
May 24, 2018 - Friends tend to have similar pain tolerance levels, study reveals
May 24, 2018 - International researchers succeed in stopping growth of mesothelioma in model animals
May 24, 2018 - Inactive people find high-intensity interval training to be as enjoyable as traditional exercise
May 24, 2018 - Now more of us can count on more time dodging the dementia bullet
May 24, 2018 - Scientists develop new test to predict woman’s risk of preterm birth
May 24, 2018 - Global healthcare access and quality improved from 2000-2016
May 24, 2018 - Virtual follow-up visits for hypertension care just as effective as in-person office visits
May 24, 2018 - New research reveals links between type 1 diabetes and mental health
May 24, 2018 - Antioxidant-enriched multivitamin may decrease respiratory illnesses in CF patients, finds study
May 24, 2018 - Antidepressant treatments increase risk of weight gain, study finds
May 24, 2018 - INSYS Therapeutics Confirms Outcome of FDA Advisory Committee Meeting on Buprenorphine Sublingual Spray
May 24, 2018 - Poor older adults with Medicaid insurance more likely to die after hospital discharge
May 24, 2018 - Early-life obesity linked to children’s lower perceptual reasoning and working memory scores
May 24, 2018 - Health and diagnostics to soon be digitalized with advent of AI
May 24, 2018 - USC researchers develop new portable device for early-stage malaria detection
May 24, 2018 - Psychologists show that depression accelerates brain aging
May 24, 2018 - Novel IR imaging offers rapid and reliable analysis of cancer tissues
May 24, 2018 - Tau mutations may serve as novel risk factor for cancer
May 24, 2018 - Sun Pharma Announces FDA Approval of Yonsa (abiraterone acetate) to Treat Metastatic Castration-Resistant Prostate Cancer
May 24, 2018 - Nurse dead in Congo as Ebola vaccination campaign starts
May 24, 2018 - Unique imaging technique identifies biomarkers of cellular damage done by diabetic retinopathy
May 24, 2018 - Study identifies key food allergy policies that parents want in schools to improve safety of kids
May 24, 2018 - Formaldehyde risk found to be higher in e-cigarettes than originally thought
May 24, 2018 - NIH commences first-in-human trial evaluating experimental treatment for Ebola
May 24, 2018 - Study finds no link between surveillance intensity and detection of recurrence or survival in CRC patients
May 24, 2018 - FDA Alert: Oral Over-the-Counter Benzocaine Products: Drug Safety Communication
May 24, 2018 - Fiber-fermenting bacteria improve health of type 2 diabetes patients
May 24, 2018 - Free e-cigarettes do not help smokers quit, money does finds study
May 24, 2018 - Higher exposure to carbon monoxide in utero increases risk of poor lung function in infants
May 24, 2018 - Neurologists identify new type of vertigo
May 24, 2018 - Scientists identify new inherited neurodevelopmental disease
May 24, 2018 - New family support program improves patient-centered care and lowers hospitalization costs
May 24, 2018 - Researchers take important step toward finding protein biomarkers during cancer surgery
May 24, 2018 - Deadly form of black lung disease found to be increasing among U.S. coal miners
Nanoparticles carrying CRISPR gene editing tools for genetic modifications

Nanoparticles carrying CRISPR gene editing tools for genetic modifications

image_pdfDownload PDFimage_print

Researchers from MIT have now developed nanoparticles that could carry CRISPR genetic editing systems deep within the cells and perform their functions on the DNA. The paper was published in the November issue of Nature Biotechnology.

In their study they used these nanoparticles with the gene editing systems to modify certain genes in mice. Until now the carriers of genetic editing systems have been modified viruses that the scientists have used for this purpose. With the development of these nanoparticles for use in this scenario, there is no need for the complicated viral based delivery systems.

In a new study, MIT researchers have developed nanoparticles that can deliver the CRISPR genome-editing system and specifically modify genes, eliminating the need to use viruses for delivery. Image Credit: MIT

In a new study, MIT researchers have developed nanoparticles that can deliver the CRISPR genome-editing system and specifically modify genes, eliminating the need to use viruses for delivery. Image Credit: MIT

In their study the researchers led by Koch Institute research scientist Hao Yin and colleagues who used these nanoparticles carrying the CRISPR systems to remove specific genes in almost 80 percent of the liver cells of the test mice. This is an unprecedented success with CRISPR ever achieved say experts. Daniel Anderson, an associate professor in MIT’s Department of Chemical Engineering and a member of MIT’s Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science (IMES) says, “What’s really exciting here is that we’ve shown you can make a nanoparticle that can be used to permanently and specifically edit the DNA in the liver of an adult animal.”

The team targeted a particular gene called the Pcsk9 that controls levels of cholesterol. When this gene is altered or mutated, it leads to a condition of impaired cholesterol control levels called dominant familial hypercholesterolemia. There are two drugs that have been approved for use in this condition. These act by inhibiting the offensive gene. These however need to be taken continuously by the patient to control the disease. Use of these nanoparticles to edit these genes and remove the mutated parts could be one-time solution to the problem feel the researchers. Other liver diseases that are mediated by genes can also be treated in future with this technique they add.

The nanoparticles the team developed carry the DNA cutting tool which is an enzyme called Cas9. It also contains a short RNA sequence that can guide the Cas9 to the specific area so that it can make the cut and edit out the mutated gene. This is not the first time that the team led by Anderson and Yin managed a delivery system without using viruses as is traditional. They developed a similar delivery system in 2014 to treat the liver disorder tyrosinemia using gene editing tool CRISPR in an adult animal. This delivery system uses a high pressure injection technique that may damage some parts of the liver. This damage was prevented by the use of this short messenger RNA that could guide the CRISPR enzyme and now the need for the high pressure injection is eliminated.

According to Anderson, it was vital that a non-viral delivery system is developed. He explained that once these viruses are used, the body can develop antibodies against it. This makes their use the next time around difficult. The body now can react against the viral vehicle making it ineffective. Yin said the team modified the short RNA that could enhance its ability to detect and guide the Cas9 and improve its activity. With this they could successfully alter 80 percent of the liver cells he added. This led to a 35 percent reduction in the total cholesterol levels of the mice.

Anderson said that the liver is a vital organ that is the “source” of several diseases. If the DNA can be reprogrammed using this technique, when the liver is functional, several diseases can be treated. The study received financial support from National Institutes of Health (NIH), the Russian Scientific Fund, the Skoltech Center, and the Koch Institute Support (core) Grant from the National Cancer Institute.

Source:

https://www.nature.com/articles/s41467-017-01466-8

32a18a44-552a-4bfa-83eb-28d4271009b6|1|5.0

Posted in: Medical Science News | Medical Research News

Tags: Antibodies, Biotechnology, Cancer, Cas9, Cholesterol, CRISPR, DNA, Dominant, Drugs, Enzyme, Gene, Genes, Genetic, Genome, Hypercholesterolemia, Liver, Liver Diseases, Nanoparticle, Nanoparticles, RNA, Tyrosinemia, Viruses

Tagged with:

About author

Related Articles