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Single CRISPR treatment can safely and stably correct genetic disease

Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.

18 Feb 2019

Study points to potential new biomarker and drug target for amyotrophic lateral sclerosis

Research led by stem cell scientists at Harvard University points to a potential new biomarker and drug target for amyotrophic lateral sclerosis, a neurological disease that is extremely difficult to diagnose and treat.

16 Jan 2019

Research may pave way for development of potent weapons against cancer

Despite significant advances in cancer research, the disease continues to exact a devastating toll. Because cancer is a disease of the body's own cells, which mutate and develop under evolutionary pressure, conventional treatments like chemotherapy and radiation often leave behind a residue of resistant cells that go on to expand and wreak havoc.

27 Nov 2018

Gene editing could be used to halt kidney disease in patients with Joubert syndrome

For the first time scientists have identified how to halt kidney disease in a life-limiting genetic condition, which may pave the way for personalized treatment in the future.

16 Nov 2018

Study: Targeting specific genomic mutation in breast cancer improves survival

Targeting a common mutation in patients with hormone receptor positive (HR+) HER2 negative (HER2-) advanced breast cancer with the alpha-specific phosphatidylinositol-3-kinase (PI3K) inhibitor alpelisib significantly improves progression-free survival, according to late-breaking results reported at ESMO 2018.

22 Oct 2018

Deciphera presents Phase 1 clinical results of DCC-2618 in patients with gastrointestinal stromal tumors

Deciphera Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, announced the presentation today of updated preliminary results from its ongoing Phase 1 clinical study of DCC-2618, the company's broad-spectrum KIT and PDGFRα inhibitor, in patients with gastrointestinal stromal tumors as a proffered paper presentation at the European Society of Medical Oncology 2018 Congress in Munich, Germany.

22 Oct 2018

New blood test may detect early-stage lung cancer when surgical intervention is not possible

Non-small-cell lung carcinoma is often fatal because most cases are not diagnosed until they are so advanced that surgical intervention is no longer possible. To improve outcomes researchers are developing a blood test to detect lung cancer earlier in the disease.

8 Oct 2018

Pfizer announces FDA approval of VIZIMPRO for first-line treatment of EGFR-mutated metastatic NSCLC

Pfizer Inc. today announced that the U.S. Food and Drug Administration has approved VIZIMPRO [vih-ZIM-pro] (dacomitinib), a kinase inhibitor for the first-line treatment of patients with metastatic non-small cell lung cancer with epidermal growth factor receptor exon 19 deletion or exon 21 L858R substitution mutations as detected by an FDA-approved test.

28 Sep 2018

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

New assay may help improve both sarcoma diagnosis and treatment

Sarcomas are rare tumors that are often misdiagnosed. Specific recurrent chromosomal rearrangements, known as translocations, can serve as essential diagnostic markers and are found in about 20 percent of sarcomas.

20 Aug 2018

New CRISPR technology may help eliminate mutated gene sequence

In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building.

17 Aug 2018

Hearing partially restored in deaf lab mice using novel therapy

A small molecule of a drug has been successfully used to treat an inherited form of hearing loss in laboratory mice.

28 Jun 2018

KFU medical departments seek to find treatments for hereditary syndromes

Newest results were showcased at the International Myology School in Moscow on 16th - 19th May 2018. KFU was represented by Junior Research Associate Mikhail Mavlikeev.

6 Jun 2018

New guidelines intend to provide template for optimal, most up-to-date DMD care

Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy-;one of nine major types of muscular dystrophy that affects males-;live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of independence, personal relationships, emotional health, and intimacy.

13 Mar 2018

Human sperm may hold potential to serve as biomarkers of future health

Human sperm may hold the potential to serve as biomarkers of the future health of newborn infants, according to a new study by a Wayne State University School of Medicine research team.

1 Dec 2017

UT Southwestern researchers develop improved gene-editing technique to correct DMD mutations

Regenerative medicine researchers at UT Southwestern Medical Center developed an improved and simplified gene-editing technique using CRISPR/Cas9 tools to correct a common mutation that causes Duchenne muscular dystrophy.

30 Nov 2017

New method of genetic testing leads to successful targeting of MET fusion in lung cancer

There are many ways a gene can be altered and there are many genes that, when altered, can cause cancer. Testing individually for each possible alteration in every cancer-related gene is not feasible as it would require hundreds of individual tests and many, many thousands of dollars.

31 Aug 2017