All Rare & Genetic Disorders articles – Page 6
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NewsCombatting HIV using gene therapy
Researchers have created a nanomedicine loaded with siRNAs, which demonstrated a 73 percent reduction in HIV replication.
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CRISPR activation: quicker diagnosis of rare genetic diseases
Using CRISPRa to activate genes in readily accessible cells provides an effective and accurate diagnosis of genetic diseases.
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Blocking the IL-6 receptor to protect tissues from CRS
The new study’s findings suggest that CRS can be treated with an IL-6 receptor antibody that has a short half-life.
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NewsInvestigating neuron-OPC synapses in live tissue
Oligodendrocyte precursor cells and the synapses they form with neurons could be relevant to many disease conditions, including cancer.
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ArticleGrowth hormone’s influence on neuropsychiatric disorders
The discovery of a mechanism involving anxiolytic effects of GH may lead to the development of new classes of anxiolytic drugs.
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NewsModel for spinal muscular atrophy using human iPSCs
A 2D neuromuscular junction model enables high-throughput screening to discover new treatments for neuromuscular diseases.
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Novel AI algorithm improves decoding accuracy of brain signals
A new deep-learning method could enhance therapeutic devices for people with neurological or mental health conditions.
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AI system develops features of complex brains
An AI system could be used to observe how physical constraints shape brains and impact people with cognitive difficulties.
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NewsPotential biomarker found for inflammatory bowel disease
A new approach called reverse metabolomics has discovered molecules that may serve as a biomarker or a therapeutic target for IBD.
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ArticleDiscovering the principles of nervous system function
Dr William Schafer, of the MRC Laboratory of Molecular Biology, focuses on the cellular and molecular mechanisms of behaviour, which is one of the fundamental problems of biology. Schafer and his colleagues have studied the nematode Caenorhabditis elegans, using a variety of approaches such as optogenetic neuroimaging and high content ...
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NewsEngineered biofilm helps to tackle infections in CF patients
A living material resembling sputum from CF patients can grow biofilms, enabling scientists to assess the effectiveness of antimicrobials.
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NewsChemotherapy success calculated by new machine learning model
Percent necrosis calculated with machine learning model for patients with osteosarcoma provides an accurate prognosis for survival.
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NewsiPSC-derived cells can augment remuscularisation of the heart
Researchers have improved the method of regenerating heart muscle after a heart attack using a combination of iPSC-derived cells.
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ArticleProtein offers promise for treatment of reproductive conditions
Scientists found that CXCR4 protein expression outside of the uterus is important for pregnancy maintenance.
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NewsPulmonary ionocytes: unexpected discovery has implications for CF
Researchers found surprising function of pulmonary ionocytes which may affect how cystic fibrosis drugs work.
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ArticleEmotional scars: stigma with female adult acne
New research presented at the European Academy of Dermatology and Venereology (EADV) Congress 2023 in Berlin, Germany, has highlighted a previously underestimated aspect of acne's impact on individuals: its profound influence on social perception.
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ArticleHPV-associated head and neck cancers identified
Human papillomavirus (HPV)-associated head and neck cancers, specifically head and neck squamous cell carcinoma (HNSCC), are on the rise worldwide. Yet, the factors contributing to these tumours and their varying responses to treatment remain poorly understood.
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NewsSickle cell anaemia: gene therapy insights for blood disorders
Cutting-edge research conducted by experts at Michigan Medicine, the Children’s Hospital of Philadelphia, and Penn Medicine is revolutionising the approach to treating blood disorders like sickle cell anaemia through innovative gene therapy methods.
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ArticleEpisode 21 - Allogeneic cell therapies and the immune system
This episode defines allogeneic cell therapy as the administration of cells to a patient for therapeutic use, that were not initially taken from that patient.
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NewsHPV vaccine cost reimbursement could hinder vaccine access
Bare minimum reimbursement rates could be why some clinics may be struggling to offer HPV vaccination in the US.


