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Category: genetics – Page 13

Long non-coding RNAs (long ncRNAs,) are a type of RNA, generally defined as transcripts more than 200 nucleotides that are not translated into protein.

Long non-coding transcripts are found in many species.

LncRNAs are extensively reported to be involved in transcriptional regulation, and epigenetic regulation.

Long non coding RNA has been proven to be associated with multiple diseases, such as cardiovascular diseases, rheumatic diseases, cancer etc.

More detailed information ons are provided in the link below.

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As the race between U.S. and Chinese biotech companies heats up, the competition is particularly fierce in one field: CRISPR gene editing.

China has rapidly emerged as a global leader in CRISPR research. While much of the initial focus in the industry was on the use of the technology to develop cancer treatments, Chinese biotech firms have since moved to apply it to test therapies for rare diseases, including sickle cell disease and inherited eye disorders.

In many areas, Chinese companies have been more aggressive, pushing into diseases that their U.S. counterparts have shied away from, including in Duchenne muscular dystrophy and herpes virus. That willingness has raised eyebrows among some executives and academics in the U.S., while exciting others who fear the American regulators and companies have been too conservative.

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Glioblastoma (GBM) is a highly aggressive and malignant brain tumor with a poor prognosis. Treatment options are limited, and the development of effective therapeutics is a major challenge. Here are some current and emerging therapeutic strategies for GBM:

Current Therapies 1. Surgery: Surgical resection is the primary treatment for GBM, aiming to remove as much of the tumor as possible. 2. Radiation Therapy: Radiation therapy is used to kill remaining tumor cells after surgery. 3. Temozolomide (TMZ): TMZ is a chemotherapy drug that is used to treat GBM, often in combination with radiation therapy. 4. Bevacizumab (Avastin): Bevacizumab is a monoclonal antibody that targets vascular endothelial growth factor (VEGF) to inhibit angiogenesis.

Emerging Therapies 1. Immunotherapy: Immunotherapies, such as checkpoint inhibitors (e.g., PD-1/PD-L1 inhibitors) and cancer vaccines, aim to stimulate the immune system to attack GBM cells. 2. Targeted Therapies: Targeted therapies focus on specific molecular pathways involved in GBM, such as the PI3K/AKT/mTOR pathway. 3. Gene Therapy: Gene therapies aim to introduce genes that can help kill GBM cells or inhibit tumor growth. 4. Oncolytic Viruses: Oncolytic viruses are engineered to selectively infect and kill GBM cells. 5. CAR-T Cell Therapy: CAR-T cell therapy involves genetically modifying T cells to recognize and attack GBM cells. 6. Small Molecule Inhibitors: Small molecule inhibitors target specific proteins involved in GBM, such as EGFR, PDGFR, and BRAF.

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Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene.

This was the first time a mutated gene has been restored to normal.

The small study of nine patients announced Monday by the company Beam Therapeutics of Cambridge, Mass., involved fixing a spelling error involving the four base sequences — G, A, C and T — in DNA. The effect was to change an incorrect DNA letter to the right one. The result was a normal gene that functioned as it should, potentially halting liver and lung damage of patients with a rare disorder.

The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely targeted infusions.

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Scientists at Berkeley Lab are unraveling the mysteries of Bennu, a 4.5-billion-year-old asteroid, using cutting-edge technology.

The asteroid harbors traces of ancient briny water, salty minerals, and even organic molecules – potential clues to life’s origins. Researchers are using X-ray and electron microscopy to analyze these space rocks at the atomic level, revealing how early planetary systems formed. Even more exciting, they’ve found amino acids.

<div class=””> <div class=””><br />Amino acids are a set of organic compounds used to build proteins. There are about 500 naturally occurring known amino acids, though only 20 appear in the genetic code. Proteins consist of one or more chains of amino acids called polypeptides. The sequence of the amino acid chain causes the polypeptide to fold into a shape that is biologically active. The amino acid sequences of proteins are encoded in the genes. Nine proteinogenic amino acids are called “essential” for humans because they cannot be produced from other compounds by the human body and so must be taken in as food.<br /></div> </div>

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A new study published in Cell Reports reveals a breakthrough discovery linking genetic variants in the gene ITSN1 to a significantly elevated risk of Parkinson’s disease, a neurodegenerative condition that affects nearly 2% of adults older than 65 years.

These findings were subsequently validated across three independent cohorts comprising more than 8,000 cases and 400,000 controls. Importantly, ITSN1 carriers trended toward earlier age of disease onset.

ITSN1 plays an important role in how neurons send messages to each other – a process called synaptic transmission – making it particularly relevant to Parkinson’s disease, a condition in which disruption of nerve signals leads to the typical symptoms of impaired gait and balance, tremors and rigidity. “We also showed in fruit flies that reducing ITSN1 levels worsens Parkinson’s-like features, including the ability to climb. We plan to extend these investigations to stem cell and mouse models,” the author said.

Interestingly, previous studies have recently implicated similar ITSN1 mutations in autism spectrum disorder (ASD). Other emerging data also have suggested an association between ASD and Parkinson’s disease, indicating that people with ASD are three times more likely to develop parkinsonism.

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Ma, W., Wang, W., Zhao, L. et al. Bone Res 13, 35 (2025). https://doi.org/10.1038/s41413-025-00416-1

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Cells function through an intricate network of proteins, each designed for specific tasks like metabolism, tissue repair, and immune defense. These proteins are built using genetic blueprints in our DNA. A process called alternative splicing enables a single gene to generate multiple mRNA transcripts — molecules carrying genetic instructions — allowing for protein diversity.

In healthy cells, this process maintains balance. Cancer cells, however, disrupt that process to fuel their unchecked growth by disabling proteins that regulate cell proliferation.

The researchers focused on a genetic element known as a poison exon. This natural “off switch” prevents the production of certain proteins by marking their RNA messages for destruction before they can be translated. Cancer cells suppress the poison exon in a key gene called TRA2β. Without this regulation, TRA2β levels rise, promoting tumor growth and making cancer cells more aggressive.

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Researchers have unveiled the first real look at a mitochondrial protein strongly linked to Parkinson’s disease, revealing key details in how its malfunction might play a critical role in the disease’s progress.

Scientists have known for more than two decades that mutations in the gene for a protein called PTEN-induced putative kinase 1 (PINK1) can trigger early-onset Parkinson’s, but the mechanisms at play have remained a mystery.

A team of scientists from the Walter and Eliza Hall Institute of Medical Research (WEHI) in Australia used advanced imaging technology to not only determine the structure of PINK1, but to show how the protein attaches to cellular power houses and how they are activated.

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