Company to Deploy Gene Editing for Alpha-1 Disease
Prime Medical will use its gene editing platform to correct the gene mutations that lead to alpha-1 antitrypsin deficiency (AATD) lung disease and liver disease.
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Company to Deploy Gene Editing for Alpha-1 Disease
Prime Medical will use its gene editing platform to correct the gene mutations that lead to alpha-1 antitrypsin deficiency (AATD) lung disease and liver disease.
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Novel Bone Marrow Transplant Can Cure Sickle Cell Disease
A novel transplant procedure using bone marrow from a “half-matched” donor is safe and curative for adults with sickle cell disease, as well as more affordable than recently approved gene therapy products for SCD.
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CRISPR-TB Blood Test Gets FDA Breakthrough Device Designation
The test uses a molecular diagnostic platform to detect Mycobacterium tuberculosis DNA in blood samples.
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‘Undeniable Link’ Between Primary Ciliary Dyskinesia and Asthma in Kids
Children with primary ciliary dyskinesia, a rare genetic disorder affecting airway function, are 22 times more likely to have asthma than children without PCD, research shows.
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CF Foundation Invests $15M in ReCode to Develop Gene Editing Therapy
The Cystic Fibrosis Foundation is funding the development of a gene editing therapy that is designed to use ReCode’s tissue-specific delivery vehicle to transport gene editing cargo to the lung cells in people with cystic fibrosis.
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AI-Powered Study to Identify Genetic Subtypes of BPD
The NIH-funded study aims to establish disease subtypes that would help predict longer-term cardiorespiratory outcomes early in the course of neonatal care.
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Genetic Risk for COPD Linked to Lower Lung Function from Childhood
A new study finds individuals with higher genetic risk for COPD show lower lung function starting in childhood.
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Does Chronic Cough Run in the Family? Study Suggests Genetic Link
Researchers found that children of parents with chronic dry cough are over 50% more likely to develop it themselves.
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First Patients Dosed in Two Phase 1 Trials for Novel Cystic Fibrosis Treatments
The phase 1 trials are evaluating two NBD1 stabilizers, SION-719 and SION-451, which are designed to improve CFTR protein function in cystic fibrosis patients.
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Scientists Correct Cystic Fibrosis Genes in Mice for Almost Two Years
The scientists developed nanoparticles that successfully edited the disease-causing gene in the lungs of a mouse model of cystic fibrosis.
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Critical DNA Regions Linked to Rare Infant Lung Disease
New research reveals how non-coding deletions in the FOXF1 gene lead to a rare, lethal infant lung disease, with implications for diagnosing and treating other pulmonary disorders.
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Mouse Lungs Successfully Generated in Rats
A combination of CRISPR/Cas9 genome editing and blastocyst complementation was used to produce mouse lungs in rats for the first time.
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Genetic Disorder Affects Macrophages, Causes Rare Lung Disease
A never-before-documented genetic disorder causes the improper functioning of macrophages, which can lead to chronic conditions in children such as pulmonary alveolar proteinosis.
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Cystic Fibrosis Foundation Funds Prime Medicine’s Gene Editing Research
The Cystic Fibrosis Foundation will provide up to $15 million to Prime Medicine for preclinical research into gene editing for cystic fibrosis.
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Why COVID-19 Spares Some and Strikes Others
In a new study, Cedars-Sinai investigators explore what factors increase susceptibility to COVID-19.
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Model Created to Decode ABCA3 Mutation’s Role in Childhood Lung Disease
Researchers demonstrate how they can now study the most common genetic cause of pediatric interstitial lung disease using stem cells generated from each patient.
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FDA Approves First CRISPR-based Therapy for Sickle Cell Disease
The FDA approved the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years and older. One therapy, Casgevy, is the first FDA-approved treatment to utilize the genome editing technology CRISPR/Cas9
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Down Syndrome Linked to Apneas, Increased Anemia
Down syndrome (DS) has been linked with medical conditions such as anemia and periods of breathing difficulties called apneas.
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Severe Sickle Cell Patients Have High Risk Tolerance for Gene Therapies
Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published in Blood Advances.
Read MoreHealth Canada OKs Trikafta for Younger Children With Cystic Fibrosis
Health Canada granted market authorization for the expanded use of Trikafta to include children with cystic fibrosis ages 2-5 years who have at least one copy of the F508del mutation in the CFTR gene.
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FDA Approves Pfizer’s Drug Combo for Specific Lung Cancer Mutation
The FDA approved the combination therapy—previously approved for certain metastatic melanoma patients—for the treatment of metastatic non-small cell lung cancer with a BRAF V600E mutation.
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Smoking Hastens Aging, Study Finds
Researchers found that smoking significantly shortens telomeres—indicators of aging and cellular repair capability—and the more cigarettes consumed, the greater the shortening effect.
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How Genes Make Us Prone to Allergies
Researchers identified how genetic differences that alter a specific protein can affect our body’s response to allergies, leading to an increased likelihood for allergic reactions that cause inflammation.
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Sleep Apnea Alters Gene Activity Patterns Over 24 Hours
The largest changes were found in lung, where intermittent hypoxia affected transcription of almost 16% of all genes, most of which were upregulated.
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