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Lilly and ProQR to expand genetic medicine development agreement

Pharmaceutical Technology

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. The post Lilly and ProQR to expand genetic medicine development agreement appeared first on Pharmaceutical Technology.

Genetics 328
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Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

XTalks

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. These mutations can vary in severity and impact on CFTR function, from complete protein dysfunction to defects in protein folding, trafficking or regulation.

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Novel genomic screening tool enables precision reverse-engineering of genetic programming in cells

Medical Xpress

Collaborative research led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center defines a novel approach to understanding how certain proteins called transcription factors determine which genetic programs will drive cell growth and maturation. The study is published in the journal Science.

Genetics 103
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Novel CSF biomarkers signal new avenues for Parkinson’s therapeutics

Pharmaceutical Technology

Last week, Nature magazine published a paper detailing an in-situ study of Parkinson’s disease (PD), showing protein structural changes in cerebrospinal fluid (CSF) between healthy individuals and Parkinson’s patients. In PD clinical research, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.

Protein 264
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Researchers discover a novel genetic disorder associated with neurodevelopmental differences

Medical Xpress

Researchers from Children's Hospital of Philadelphia (CHOP) and Princeton University have discovered a novel genetic disorder associated with neurodevelopmental differences. The study "Abrogation of MAP4K4 protein function causes congenital anomalies in humans and zebrafish" is published in Science Advances today, April 26.

Genetics 100
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Japanese-European research team discovers novel genetic mitochondrial disorder

Scienmag

Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.

Genetics 105
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Genetic eraser: Newly developed technology precisely and rapidly degrades targeted proteins

Scienmag

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].

Protein 98