Gene Therapy Treatment Allows 8-Year-Old to See Stars for the First Time

Sam is 8 years old and is the first Canadian to go through gene therapy for his form of blindness. Sam suffers from a rare disorder known as retinitis pigmentosa; a form of progressive blindness caused by genetic retinal degeneration that results from mutations in the RPE65 gene.

CRISPR can Cause Serious Side Effects in Human Embryos

A recent study revealed that instead of addressing genetic mutations, CRISPR prompted cells of human embryos to lose entire chromosomes. The study administered Crispr-Cas9 technology to cells to repair a mutation that causes hereditary blindness, but instead, appeared to wreak havoc in almost half of the specimens examined by the researchers.

Coral Gene Editing Study Reveals Gene for Heat Tolerance

An international research project used CRISPR-Cas9 gene-editing technology to examine the heat tolerance of Great Barrier Reef coral with the results set to guide efforts in combatting the effects of climate changes. The study used CRISPR to make precise, targeted changes to the genome of coral. Using this approach, the team demonstrated the importance of a particular gene on heat tolerance in the coral Acropora millepora.

Safer, More Targeted Delivery Method for CRISPR Gene Therapy

Biomedical engineers from the University of New South Wales and other institutions discovered that liposomes—commonly used in pharmacology to encapsulate drugs or genes—can be triggered by light to release CRISPR gene therapy in a specific site in the body, and the method could prove to be safer and more direct than current methods. Once the liposomes are triggered by light, they eject the CRISPR contents, which then go to work looking for genes of interest.

CRISPR-Based Genome Editing System Destroys Cancer Cells🧬🦠👾

Recent studies at Tel Aviv University have demonstrated the effectiveness of CRISPR-Cas9 in treating metastatic cancers. The researchers developed a novel lipid nanoparticle-based delivery system that specifically targets cancer cells and destroys them via genetic manipulation.

HIV-Like Virus Edited Out of Primate Genome Using CRISPR 🦠🐒🧬

Scientists from the Lewis Katz School of Medicine at Temple University have successfully edited SIV – a virus closely related to HIV, the cause of AIDS – from the genomes of non-human primates. The breakthrough brings researchers closer than ever to developing a cure for human HIV infection.

‘Off Switch’ Offers Promising Fix to CRISPR’s Problem with Unwanted Genetic Changes 🚫✂️🧬

A group of researchers from Hiroshima University have developed a potential fix to CRISPR-Cas9’s problem with unwanted genetic changes using a method that allows them to turn off gene-editing until it reaches key cell cycle phases where more accurate repairs are likely to happen. In their study, they demonstrated more precise gene-editing and suppressed unintended genetic deletions, insertions, or mutations called off-target effects.

CRISPR-Cas9 Proves Successful for Two Patients with Common Blood Disorders 🧬🩸💉

CRISPR and another genetic strategy have proved that cures are possible for many people born with sickle cell disease and another serious blood disorder known as beta-thalassemia, a first for the genome editing technology. More than a year later, both patients had clinically improved and no longer needed blood transfusions.

Genetically Modified Tomatoes Produce Medicine to Treat Parkinson’s Disease 🍅🧬🧠

Researchers from The John Innes Centre in the UK engineered tomatoes to produce L-DOPA to serve as a more accessible form of the medication. They inserted a gene from beetroots that produces L-DOPA in the process of producing pigments into tomatoes hoping to achieve higher levels of L-DOPA to extract later and purify in later stages.

CRISPR-Based Test for COVID-19 Uses a Smartphone Camera 🧬🦠📱

One of the major hurdles to combating the COVID-19 pandemic across the country is the availability of mass rapid testing. A team of scientists from Gladstone Institutes, University of California, Berkeley, and University of California, San Francisco have outlined the technology for a rapid, one-step mobile test capable of providing test results from your phone in under 30 minutes using CRISPR.

CRISPR Reveals How Coral Reefs Respond to Heat Stress 🧬🌊🌡

Researchers from Carnegie Institution for Science used CRISPR/Cas9 gene editing tool to investigate the genetic mechanisms behind the process of coral bleaching.

New Gene-Editing Technology Capable of Altering Several Thousand Genes at Once to Determine Their Impacts 🧬🔬✂️

The new technique, called CiBER-seq, can rapidly determine all the DNA sequences in the genome that regulate the expression of a specific gene. Based on CRISPR genome editing, the new technology developed by researchers at the University of California, Berkeley links targeted, genome-wide genetic changes with a deep-sequencing readout that quantitatively measures the expression phenotype induced by each edit.

Genetic Engineering Without Unwanted Side Effects Helps Fight Parasites 🧬🧫🦠

Modified CRISPR-Cas9 gene editing scissors are allowing researchers to make alterations to the genetic material of single-cell organisms that are indistinguishable from natural mutations. This approach makes it possible to develop a harmless experimental live vaccine for the widespread parasite known as Toxoplasma gondii.

CRISPR Cures Progeria in Mice, Inspiring Plans for One-Time Therapy to Treat the Disease 🧬🧫🦠

CRISPR gene editing has been used to more than double the lifespan of mice engineered to have the premature aging disease known as progeria. Researchers from the Broad Institute used base editing to alter a single misspelled pair of “letters” among 3 billion in the DNA of cells taken from children with progeria. If the results are confirmed in human trials, the method could prove to be a true genetic cure with a single injection, and not a drug children will have to take all their lives.

CRISPR-Based Genetic Screen Identifies KAT7 as a Gene that Drives Aging 🧬🔬👵🏼

Cellular senescence is known to promote aging, however, many of the mechanisms controlling this process remain poorly understood. Researchers conducted a genome-wide CRISPR-based screen to identify genes that could affect cellular senescence, where they identified the gene KAT7. Switching this gene off extended the lifespan in mice aging normally and in mice with diseases known to cause premature aging.

Scientists Use CRISPR to Program Living Cells to Store Data 🧬🧫💽

Researchers from the Wang Lab in Columbia University Medical School developed a strategy to encode digital data into the genomes of living cells without in-vitro DNA synthesis. Data recording in vivo by electrical stimulation (DRIVES) aims to use electrical signals to essentially “copy and paste” code directly from a computer to living cells. 

Paralyzed Mice Walk Again with Designer Cytokine 🧬 🐁 🧠

A research team at Ruhr-University Bochum succeeded in getting mice to walk again, after a complete cross-sectional injury using gene therapy. The nerve cells produced the curative protein themselves.

Plant Genome Editing Using a Newly Derived CRISPR-Cas9 Tool🧫🧬🔬

The first CRISPR-Cas9 tool that started the gene-editing trend was linked to targeting a certain short sequence of DNA referred to as a PAM sequence, which CRISPR normally uses to recognize where to make their molecular cuts in DNA. Nevertheless, the new SpRY variant can move past these conventional PAM sequences in ways which impossible earlier.

Zebrafish Used to Study Spinal Deformities in Humans🔬🐟👨‍🔬

The embryonic tissue of a zebrafish is transparent, which enables researchers to see what is going on inside. Normally, zebrafish’s spine patterns give the appearance of stripes. It’s development is precisely controlled by segmentation genes. In this study, zebrafish embryos were used to study a gene mutation which is the cause behind scoliosis, a sideways curvature of the spine that occurs in humans just before puberty. Zebrafish and humans share about 70% of the same genes.

Genetically Modified Mosquitoes – Key to Stopping Zika Virus🦟🦠🧬

Researchers at the University of Missouri genetically modified mosquitoes to be resistant to Zika virus. Zika is spread to people primarily through the bite of an infected Aedes species mosquito. In 2016, the World Health Organization called the Zika virus epidemic a ‘public health emergency of international concern’ due to the virus causing birth defects for pregnant women in addition to neurological problems.

TALEN Gains Traction In Gene Editing After Outperforming CRISPR 🧬🔬🧑‍🔬

Researchers from University of Illinois at Urbana-Champaign (UIUC) tested the gene editing efficiency of TALEN and CRISPR on heterochromatin, allowing some researchers to argue that it can be a better technique for hard-to-edit regions of the genome. 

CRISPR Offers Powerful Antiviral Protection & Detection of RNA Viruses🧬🔬🦠

Scientists believed that CRISPR effectors could be repurposed to help in defending human cells against both DNA & RNA viruses. Previous studies have shown that CRISPR-Cas13 possesses the ability to efficiently target and to cleave the RNA in several model systems, including the human cells.

CRISPR Technology Targets the Human Genome’s Complex Code🧬🧫🔬

A new genome-editing tool has been created by scientists at Rice University using CRISPR technology that aims at the nucleus’s sustaining players that carry out DNA packaging and help gene expression. This opens frontiers to novel treatment approaches for cancer and other diseases.

Researchers Create Lab-Grown Brain Using CRISPR 🧫🧠🧬

Researchers from the University of Basel in Switzerland created small brain-like “organoids” with gene variants from human relatives, Neanderthals and Denisovans. The organoids were made by engineering human stem cells with genes that were common to Neanderthals and humans.