Gene Editing News and Research

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Gene therapy for degenerative retinal disease has no safety concerns but fails to improve vision loss

A 28-patient phase 1 gene therapy clinical trial for the degenerative retinal disease Leber hereditary optic neuropathy (LHON) found no significant safety concerns; however, treatment failed to improve or slow vision loss, with even the highest dose. LHON affects the optic nerve, which carries visual signals from the light-sensing retina to the brain.

1 Jun 2022

Aging does not influence liver renewal, research shows

The liver has a unique ability to regenerate after damage. However, it was unknown whether this ability decreases as we age.

31 May 2022

Researchers identify a gene that plays vital role in the innate human immune system

Cedars-Sinai investigators have identified a gene that plays an essential role in the innate human immune system. The gene, NLRP11, helps activate the inflammatory response that tells the body's white blood cells to go on the attack against a foreign presence.

27 May 2022

Penn biologists show evidence of a two-sided genomic arms race involving repetitive DNA

Biological arms races are commonplace in nature. Cheetahs, for example, have evolved a sleek body form that lends itself to rapid running, enabling them to feast upon similarly speedy gazelles, the fastest of which may evade predation.

27 May 2022

Understanding transposons provides a new starting point to generate more powerful gene editing tools

CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases.

27 May 2022

UH experts in cloning and stem cell science take the first step toward unraveling cystic fibrosis

Two nationally recognized experts in cloning and stem cell science from the University of Houston are taking the first step toward limiting the consequences of chronic inflammation in cystic fibrosis (CF) by identifying the source of this persistent and enigmatic inflammation in CF lungs.

24 May 2022

Gene-edited tomatoes could be a simple solution to address vitamin D insufficiency

Tomatoes gene-edited to produce vitamin D, the sunshine vitamin, could be a simple and sustainable innovation to address a global health problem.

23 May 2022

The promise of RNA medicine is targeted and personalized

The development of messenger RNA (mRNA) vaccines against the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has only increased the impetus in this field.

11 May 2022

Deficiency of YWHAZ gene alters neurodevelopmental process in zebrafish

An article published in the journal Molecular Psychiatry reveals the molecular mechanisms to explain how the YWHAZ gene –related to psychiatric and neurological disorders such as autism and schizophrenia– can alter the neurodevelopmental process.

10 May 2022

bit.bio launches Huntington’s disease human cell model

First neurodegenerative disease model launched from bit.bio's new ioDisease Model portfolio.

From bit.bio 10 May 2022

New method increases efficiency of gene editing while minimizing DNA deletion sizes

Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

2 May 2022

Study offers critical clues into longstanding mystery of deep sleep

Healthy sleep is a basic physiologic need. In its absence, a myriad of processes in the body can go terribly awry. Chronic sleep problems have been linked to mental health disorders, cardiovascular disease, type 2 diabetes, and obesity, among other conditions.

28 Apr 2022

Genetic cause of lupus identified

An international team of researchers has identified DNA mutations in a gene that senses viral RNA, as a cause of the autoimmune disease lupus, with the finding paving the way for the development of new treatments.

27 Apr 2022

Gene discovery could pave the way for new and more effective treatments for lupus

Researchers from The Australian National University have identified a gene called TLR7 that, when over-activated, is responsible for causing lupus, an autoimmune disease that can be life-threatening in severe cases.

27 Apr 2022

Researchers develop new platform capable of mitochondrial DNA editing

Researchers from the Center for Genome Engineering within the Institute for Basic Science developed a new gene-editing platform called transcription activator-like effector-linked deaminases, or TALED.

25 Apr 2022

Advancements in HIV science

As far as viruses go, human immunodeficiency virus (HIV) is quite simple. About 100,000 times smaller than a red blood cell, the virus expresses just a dozen proteins, yet it can establish a lifelong infection that, if left untreated, causes acquired immunodeficiency syndrome (AIDS) and death.

20 Apr 2022

Novel targets for treating sorafenib-resistant hepatocellular carcinoma found using CRISPR-Cas9 screening

This study is led by Xiujun Cai (Department of General Surgery, Sir Run-Run Shaw Hospital, Zhejiang University School of Medicine). Professor Xiujun Cai's research team screened the whole genome of hepatocellular carcinoma cells (HepG2) through CRISPR / cas9 system under sorafenib treatment, and screened the dominant gene in sorafenib resistance.

20 Apr 2022

Gene Editing News and Research

Gene therapy for degenerative retinal disease has no safety concerns but fails to improve vision loss

Aging does not influence liver renewal, research shows

Researchers identify a gene that plays vital role in the innate human immune system

Penn biologists show evidence of a two-sided genomic arms race involving repetitive DNA

Understanding transposons provides a new starting point to generate more powerful gene editing tools

UH experts in cloning and stem cell science take the first step toward unraveling cystic fibrosis

Gene-edited tomatoes could be a simple solution to address vitamin D insufficiency

The promise of RNA medicine is targeted and personalized

Deficiency of YWHAZ gene alters neurodevelopmental process in zebrafish

bit.bio launches Huntington’s disease human cell model

New method increases efficiency of gene editing while minimizing DNA deletion sizes

Study offers critical clues into longstanding mystery of deep sleep

Genetic cause of lupus identified

Gene discovery could pave the way for new and more effective treatments for lupus

Researchers develop new platform capable of mitochondrial DNA editing

Advancements in HIV science

Novel targets for treating sorafenib-resistant hepatocellular carcinoma found using CRISPR-Cas9 screening

Recent advances in non-viral nano-vector delivery of CRISPR/Cas9 genome editing payloads

UNSW research improves fundamental understanding of the mechanism behind sickle cell disease

Powerhouse-pruning protein may also help make energy rapidly available for growing new blood vessels

Industry focus eBook - Cell and gene therapy (2nd edition) eBook

Advancing Intracellular Delivery with LumiPore®

How ELRIG Is Helping Mid-Career Scientists Navigate Leadership, Career Change and Redundancy

The role of UV-Vis in biopharma QC

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