Gene Editing News and Research

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Sartorius BIA Separations and Exopharm Sign Joint Research Agreement to Develop Integrated Technology

Sartorius BIA Separations (BIA), a leading bio-chromatography company, now part of the international life science group Sartorius, and Exopharm Ltd. (ASX:EX1), a company at the forefront of transformative medicines using exosomes, or extracellular vesicles (EVs), today announced they had entered into a formal collaboration.

From Sartorius 23 Nov 2022

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed.

22 Nov 2022

Discovery could lead to new mantle cell lymphoma drugs

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.

22 Nov 2022

Rice bioengineer receives support for brain cancer research

Kevin McHugh, an assistant professor of bioengineering at Rice University's George R. Brown School of Engineering, has earned a Distinguished Scientist Award for 2022 from The Sontag Foundation, which helps launch the careers of scientists expected to make a significant impact in the fight against brain cancer.

21 Nov 2022

Gene mutation associated with autism causes an overstimulation of brain cells

Scientists looking to understand the fundamental brain mechanisms of autism spectrum disorder have found that a gene mutation known to be associated with the disorder causes an overstimulation of brain cells far greater than that seen in neuronal cells without the mutation.

21 Nov 2022

MIT researchers develop new technique for genome-wide studies of the liver

The liver’s ability to regenerate itself is legendary. Even if more than 70 percent of the organ is removed, the remaining tissue can regrow an entire new liver.

21 Nov 2022

Study reveals mechanism by which malignant cells shut down anti-cancer immune responses

A Ludwig Cancer Research study has revealed a single protein expressed at high levels by cancer cells across a broad range of malignancies that erects a multifaceted barrier to anti-cancer immune responses in mouse models of cancer and so shields tumors from immune detection and destruction.

20 Nov 2022

What are the current challenges and future directions of mRNA nanomedicine?

Researchers presented challenges related to the clinical translation of messenger ribonucleic acid (mRNA) technology, recent innovations and future directions to improve the clinical efficacy of mRNA nanomedicine.

15 Nov 2022

Behind the Scenes of the Oncology & Precision Medicine Conferences 2022

During the conference, we caught up with some of the speakers to learn more about their work, what they were presenting, and their thoughts about the future of Oncology and Precision Medicine.

15 Nov 2022

New CRISPR approach is a leap forward in developing personalized treatment for cancer

For the first time, scientists have used CRISPR technology to insert genes that allow immune cells to focus their attack on cancer cells, potentially leaving normal cells unharmed and increasing the effectiveness of immunotherapy.

10 Nov 2022

Novel branched ionizable lipid greatly increases the efficiency of mRNA delivery

Messenger RNA (mRNA) are biological molecules that transfer the information coded by genes in the nucleus to the cytoplasm for protein synthesis by ribosomes.

10 Nov 2022

Tissue nanotransfection shows promise as a treatment for traumatic muscle loss

Technology developed by researchers at the Indiana University School of Medicine that can change skin tissue into blood vessels and nerve cells has also shown promise as a treatment for traumatic muscle loss.

9 Nov 2022

Novel gene editing therapy reduces circulating TTR protein levels in patients with ATTR amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022.

7 Nov 2022

Beyond vaccines: emerging technologies capable of unlocking the promise of biologically targeted mRNA therapeutics

Researchers reviewed technology advancements that will unlock the promise of biologically targeted messenger ribonucleic acid (mRNA) therapeutics beyond vaccines.

7 Nov 2022

New control system could be used to finely tune the production of useful proteins

Using an approach based on CRISPR proteins, MIT researchers have developed a new way to precisely control the amount of a particular protein that is produced in mammalian cells.

1 Nov 2022

Researchers use CRISPR-edited T cells to treat seriously ill children with resistant leukaemia

Researchers at Great Ormond Street Hospital for Children (GOSH) and UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) have used CRISPR/Cas9 technology to engineer donor T cells to try to treat seriously ill children with resistant leukaemia, who had otherwise exhausted all available therapies.

27 Oct 2022

Discovery points to a new understanding of Stargardt disease progression

Using a new stem-cell-based model made from skin cells, scientists found the first direct evidence that Stargardt-related ABCA4 gene mutations affect a layer of cells in the eye called the retinal pigment epithelium (RPE).

27 Oct 2022

Pioneering gene editing technique can repair faulty T cells in patients with CTLA-4 insufficiency

A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and mice, led by UCL scientists.

27 Oct 2022

New studies point to a broadly applicable treatment for neurodegenerative diseases

Two new studies from Washington University School of Medicine in St. Louis support development of a broadly applicable treatment for neurodegenerative diseases that targets a molecule that serves as the central executioner in the death of axons, the wiring of the nervous system.

26 Oct 2022

Butterfly wing pattern ground plan is manipulated by non-coding regulatory DNA, study says

Butterfly wing patterns have a basic plan to them, which is manipulated by non-coding regulatory DNA to create the diversity of wings seen in different species, according to new research.

21 Oct 2022

Gene Editing News and Research

Sartorius BIA Separations and Exopharm Sign Joint Research Agreement to Develop Integrated Technology

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

Discovery could lead to new mantle cell lymphoma drugs

Rice bioengineer receives support for brain cancer research

Gene mutation associated with autism causes an overstimulation of brain cells

MIT researchers develop new technique for genome-wide studies of the liver

Study reveals mechanism by which malignant cells shut down anti-cancer immune responses

What are the current challenges and future directions of mRNA nanomedicine?

Behind the Scenes of the Oncology & Precision Medicine Conferences 2022

New CRISPR approach is a leap forward in developing personalized treatment for cancer

Novel branched ionizable lipid greatly increases the efficiency of mRNA delivery

Tissue nanotransfection shows promise as a treatment for traumatic muscle loss

Novel gene editing therapy reduces circulating TTR protein levels in patients with ATTR amyloid cardiomyopathy

Beyond vaccines: emerging technologies capable of unlocking the promise of biologically targeted mRNA therapeutics

New control system could be used to finely tune the production of useful proteins

Researchers use CRISPR-edited T cells to treat seriously ill children with resistant leukaemia

Discovery points to a new understanding of Stargardt disease progression

Pioneering gene editing technique can repair faulty T cells in patients with CTLA-4 insufficiency

New studies point to a broadly applicable treatment for neurodegenerative diseases

Butterfly wing pattern ground plan is manipulated by non-coding regulatory DNA, study says

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