Gene Therapy

CRISPR Based Therapy Shows Promise for Blood Disorders

The first gene editing clinical trial sponsored by US companies has released data to STAT news showing promising results using a CRISPR based therapy. Sickle cell anaemia and beta thalassemia patients required no further treatments after one appointment.

New CRISPR-Cas9 Protein can Increase Gene Editing Efficiency

A new version of the CRISPR-Cas9 protein has been developed that can increase the targeting efficiency of the gene editing process. Currently the CRISPR Cas-9 system is the most widely used gene editing technique. However, concerns about CRISPR editing genes other than the ones it is targeted to have hindered its introduction into gene-therapy treatments […]

NIH, Gates Foundation Commit $200M to Gene-Based Cures for Sickle Cell and HIV

  Press Release from National Institute of Health  The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates Foundation will also invest $100 million toward this goal. The […]

New Prime Editing Tool Will Revolutionise Gene Therapy

A new gene editing tool known as Prime Editing has been developed that could reverse the harmful mutations in over 89% of genetic diseases. Building on the CRISPR Success Currently the CRISPR Cas-9 system is the most widely used gene editing technique. However, concerns about CRISPR editing genes, other than the ones it is targeted […]

Danger of CRISPR Off-Target Mutations Quantified

CRISPR gives us the ability to precisely edit genes within cells, and the technique holds enormous potential for transforming the gene therapy space. However, concerns about CRISPR editing genes other than the ones it is targeted to have hindered its introduction into patient treatments. Known as off-target mutations, these unintended gene edits caused by CRISPR […]

Shareable Science: Hachimoji – A New Genetic Language?

Our guest contributor Dr Neil Lamb continues his fortnighty Shareable Science Blog with an extract from the annual guidebook ‘A Year in Genetics as Told by Tomorrow’s Textbooks‘. Neil is the Vice President for Educational Outreach at the HudsonAlpha Institute for Biotechnology and Shareable Science will explore how genetics is relevant to people in their […]

Synthetic DNA: An Interview with Michael Kamdar, CEO Molecular Assemblies

Molecular Assemblies recently announced a $12.2M series A financing to advance their enzymatic DNA synthesis technology. We talked to Michael Kamdar, the CEO and president of Molecular Assemblies, to discuss how enzymatic DNA synthesis could revolutionise biotechnology. FLG: Can you introduce Molecular Assemblies? MK: Molecular Assemblies is a synthetic biology company founded in 2013, and […]

Muscular Dystrophy Oligonucleotide Treatment Shows Promise in Pre-Clinical Trials

An oligonucleotide treatment for muscular dystrophy has shown promising results in pre-clinical trials. It is estimated that the new treatment could be effective for over half of muscular dystrophy patients. Muscular dystrophy is a hereditary condition characterised by a progressive weakening and wasting of the muscles. It is caused by a malfunction in the gene […]

First CRISPR Therapy to Treat HIV Safe but Ineffective

A CRISPR therapy that attempted to cure a man of HIV has failed, but proved safe for the patient. The patient was diagnosed with HIV/AIDs and acute lymphoblastic leukemia. He was being treated with chemotherapy for the leukemia and anti-viral drugs for HIV, which reduce the amount of virus in the blood but do not […]

Cancer Cells Can Cannibilise to Survive Chemotherapy

Cancer cells can consume neighbouring cells to survive chemotherapy, new research shows. Doxorubicin is a common chemotherapy drug that works by selectively damaging the DNA of cancer cells. However, multiple cancer cells have been observed to survive doxorubicin treatment to allow a relapse of the disease. These cancer cells can enter a form of dormancy, […]

Webinar: Advances in Improving HDR Efficiency for CRISPR Experiments

Front Line Genomics hosted the webinar Improved Methods for CRISPR Homology-Directed Repair Using Alt-R HDR Enhancer and ssDNA Donors with Optimized Design, which you can watch on demand for free here. The webinar described how to overcome some of the bottlenecks to using the CRISPR-Cas9 for genome manipulation and ensure its efficacy in a clinical […]

First CRISPR Law Prevents DIY Gene Therapy

The first law to regulate the use of genome editing technology has been passed in California.  Senator Ling Ling Chang authored the bill that requires sellers of gene therapy kits to include a label, and a notice prior to sale, stating that the CRISPR kits are not intended for self-administration. The bill is targeting ‘DIY […]

Sticky DNA Traps Cause Gallstone Formation

Sticky DNA traps produced by immune cells enable gallstones to stick together and grow, new research has revealed. Gallstones are stones that form in the gall bladder when the fats and minerals found in bile harden into a crystalline structure. Gallstones can cause inflammation and infections, depending on their size. Researchers investigated gallstones in patients […]

Damaged Neural Connections Repaired with Nanoparticles

A protein produced by specific cells in the brain has been shown to repair damaged brain networks. This discovery could allow a genetically engineered treatment for neurogenerative disorders such as Alzheimer’s. A key hallmark of neurogenerative diseases is the decay of the connections between neurons in the brain. If these connections could be restored it […]

Genes that Allow Fish to Re-grow Limbs also Present in Humans

The genes that enable a fish to re-grow limbs have been found to be present in humans, new research has found. This insight could help develop regenerative treatments in humans. Many species can regenerate limbs after amputation including fish, salamanders and geckos. This can be used as a mechanism to escape predators and heal from […]