German Center for Lung Research (DZL)’s Post

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

📃Scientific paper: Clinical Application of Circulating MicroRNAs in Parkinson's Disease: The Challenges and Opportunities as Diagnostic Biomarker Abstract: Discovery of evolutionarily conserved, nonprotein-coding, endogenous microRNAs has induced a paradigm shift in the overall understanding of gene regulation. Now, microRNAs are considered and classified as master regulators of gene expression as they regulate a wide range of processes – gene regulation, splicing, translation and posttranscriptional modifications. Besides, dysregulated microRNAs have been related to many diseases, including Parkinson's and related disorders. Several studies proposed that differentially expressed microRNAs as a potential biomarker. So far, there is no accepted clinical diagnostic test for Parkinson's disease based on biochemical analysis of biological fluids. However, circulating microRNAs possess many vital features typical of reliable biomarkers and discriminates Parkinson's patients from healthy control with much higher sensitivity and specificity. Though they show tremendous promise as a putative biomarker, translating these research findings to clinical application is often met with many obstacles. Most of the candidate microRNAs reported as a diagnostic biomarker is not organ-specific, and their overlap is low between studies. Therefore this review aimed to highlight the challenges in the application of microRNA in guiding disease discrimination decisions and its future prospects as a diagnostic biomarker in Parkinson's Disease. Continued on ES/IODE ➡️ https://etcse.fr/FM ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

bluebird bio Secures Second Big Outcomes-Based Coverage Contract for #Lyfgenia - BioSpace A month after winning the FDA’s approval for its sickle cell disease gene therapy Lyfgenia (lovotibeglogene autotemcel), bluebird bio has secured its second outcomes-based coverage deal, the biotech disclosed Thursday in an SEC filing. The agreement pushes the cumulative total of covered U.S. patients for Lyfgenia to approximately 200 million people, the company noted in its filing. Bluebird previously signed a similar outcomes-based agreement in December 2023, a week after Lyfgenia’s FDA approval. The company did not name the payer but said that it represents approximately 100 million covered patients in the U.S. At the time, bluebird also revealed in an SEC filing that it was in advanced talks with other large commercial payers and with more than 15 Medicaid agencies, representing some 80% of sickle cell disease (SCD) patients in the U.S. Bluebird said it will provide more details regarding the commercial launch of Lyfgenia at next week’s 42nd annual J.P. Morgan Healthcare Conference. Lyfgenia is a gene therapy that delivers a functional copy of the β-globin gene directly into the blood stem cells, allowing patients to produce their own anti-sickling hemoglobin and reducing the overall proportion of sickled hemoglobin. https://lnkd.in/e2GBGSpB

Exciting news in healthcare access! Sickle cell disease (SCD) will lead the U.S.'s new Cell and Gene Therapy (CGT) Access Model, targeting broader access to critical therapies for vulnerable populations. Gene therapies, among the world's most expensive treatments, pose significant accessibility challenges. The Centers for Medicare & Medicaid Services (CMS) will collaborate with states and manufacturers to expand access to gene therapies for SCD. Launching in January 2025, the CGT Access Model offers states participation options until January 2026. CMS introduced three new testing models last year, including the CGT Access Model, aimed at lowering drug costs and enhancing accessibility to life-altering therapies. This program enables state Medicaid agencies to authorize CMS to coordinate outcomes-based agreements with manufacturers for specific cell and gene therapies. With the FDA approving two gene therapies for SCD in December, each carrying substantial price tags, the need for accessibility becomes more pressing. Approximately 50% to 60% of SCD patients in the U.S. are enrolled in Medicaid, with healthcare costs nearing $3 billion annually. SCD, disproportionately affecting Black Americans, lacks adequate treatment options, with over 100,000 Americans affected. The CGT Access Model, part of the Administration's mission to reduce drug costs, responds to President Biden's directive to explore models enhancing therapy access. Source: https://lnkd.in/e3mG646T

On this #rarediseaseday2024, let's take a moment to recognize the millions of individuals worldwide who are impacted by rare diseases. Did you know that a rare disease affects fewer than 0.050% of the population, or approximately 1 in 2,000 people? These complex and diverse conditions can present many challenges for patients and their families. However, there is hope on the horizon. In 2023, we saw significant advancements in gene therapy, including the approval of CASGEVY™ for the treatment of sickle cell disease and beta thalassemia in patients aged 12 and above. Additionally, a 13-year-old girl became the first reported patient in the world to receive base-edited T-cells to treat rare childhood cancer T-cell acute lymphoblastic leukemia. And in September, the world's first novel CRISPR-Cas13 RNA-editing therapy was announced for the treatment of neovascular age-related macular degeneration. As we continue to work towards finding treatments and cures for rare diseases, it's important to recognize the impact they have on individuals and their families. I am inspired daily by the science of gene therapies, and most of all, the scientists at work. At Waters Corporation and Waters | Wyatt Technology, we are committed to accelerating this research and enabling our customers to achieve their goals. To learn more about the latest clinical highlights in gene editing, check out the link below. https://lnkd.in/eDX7GsRC #RareDiseaseDay #RareDiseases #crispr #geneediting #biologics #genetherapy