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388 results about "Cardiomyopathy" patented technology

An acquired or inherited disease of the heart muscle which makes it difficult for the heart to pump blood to other parts of the body.

A monoclonal antibody combination for detecting cat NT-proBNP protein and its application

This invention belongs to the field of biological detection technology, specifically relating to a monoclonal antibody combination for detecting feline NT-proBNP protein and its applications. The monoclonal antibody combination of the present invention comprises monoclonal antibodies 3A6 and 5H5. These two antibodies possess unique heavy and light chain variable region complementarity-determining regions, respectively, and their amino acid sequences have been fully defined (SEQ ID NOs. 1-12). This antibody combination can specifically recognize and efficiently bind to feline NT-proBNP protein, making it suitable for the development of a highly sensitive and specific diagnostic tool for feline cardiomyopathy. By constructing a double-antibody sandwich ELISA assay system, the minimum detection limit can reach 100 pg / mL, and it exhibits excellent specificity and stability.
Owner:BEIJING SUBENYUANHE BIOTECHNOLOGY CO LTD

Therapeutic effect evaluation method for virtual resection operation of hypertrophic obstructive cardiomyopathy

The invention relates to a hypertrophic obstructive cardiomyopathy virtual resection operation curative effect evaluation method based on individualized computational fluid mechanics, which can give visual postoperative hemodynamic indexes, predict postoperative curative effects, find an optimal resection scheme and realize preoperative accurate intelligent evaluation. Comprising the following steps of: acquiring coronary artery enhanced computed tomography image data, acquiring a coronary artery enhanced computed tomography image of a patient, and outputting and storing the coronary artery enhanced computed tomography image in a DICOM (Digital Imaging and Communications in Medicine) format; preprocessing image data, and segmenting and reconstructing a left ventricle-outflow tract structure three-dimensional model; setting a virtual excision scheme at the fleshy part; constructing a surgical resection model, and importing the model into finite element analysis software for solving; and the virtual postoperative curative effect is visualized. Verification and curative effect prediction of a patient excision scheme can be realized, and the operation success rate of a hypertrophic obstructive cardiomyopathy patient is improved.
Owner:THE FOURTH AFFILIATED HOSPITAL OF CHINA MEDICAL UNIV

Diabetic cardiomyopathy clinical prediction model, establishment method and application thereof

The invention discloses a diabetic cardiomyopathy clinical prediction model, an establishment method and application thereof. The method includes the steps that clinical data of type II diabetes patients meeting the containing and discharging standard are collected based on public health data and processed, a random forest RF prediction model is established, the random forest RF prediction model is trained and evaluated to finally establish the random forest RF prediction model, the model accuracy is 0.889, the sensitivity is 0.896, the specificity is 0.880, and the recall rate is 0.896. The model has higher prediction accuracy and robustness, and can be used for an intuitive and convenient webpage calculator for early diagnosis of the diabetic cardiomyopathy, so that a clinician can easily predict the risk of the diabetic cardiomyopathy, resources are allocated according to a prediction result, and waste of medical resources is reduced.
Owner:FIRST AFFILIATED HOSPITAL OF KUNMING MEDICAL UNIV

Dilated cardiomyopathy zebra fish model and construction method and application thereof

The invention provides a dilated cardiomyopathy zebrafish model and a construction method and application thereof, and belongs to the technical field of biology.The dilated cardiomyopathy zebrafish model is constructed by overexpressing miR-16-5p in myocardial cells of zebrafish, and the sequence of miR-16-5p is shown as SEQ ID NO.1; the invention further provides a construction method of the dilated cardiomyopathy zebra fish model. The invention constructs the dilated cardiomyopathy zebrafish model of overexpression miR-16-5p in myocardial cells for the first time, and the dilated cardiomyopathy zebrafish model can be used for screening drugs for resisting dilated cardiomyopathy.
Owner:QILU UNIVERSITY OF TECHNOLOGY (SHANDONG ACADEMY OF SCIENCES) +1

Use of alisol-b23-acetate in prevention or treatment of hypertrophic cardiomyopathy

Disclosed in the present invention is a use of alisol-B23-acetate (AB23a) in the preparation of a drug for preventing or treating hypertrophic cardiomyopathy and diseases caused by the hypertrophic cardiomyopathy. The present invention provides for the first time a use of AB23a in the prevention or treatment of hypertrophic cardiomyopathy. According to the present invention, use of AB23a in direct in vitro treatment of cardiomyocytes derived from directed differentiation of human embryonic stem cells revealed that AB23a-based in-vitro treatment can significantly inhibit hypertrophic phenotypes of the human embryonic stem cells-cardiomyocytes; using an AB23a-containing feed to feed mice with hereditary hypertrophic cardiomyopathy caused by a gene mutation revealed that AB23a prominently relieves pathological myocardial hypertrophy of the mice and improves the cardiac function; AB23a can be used for preparing a drug for resisting hereditary hypertrophic cardiomyopathy, offering a novel way and means for treating hypertrophic cardiomyopathy; and AB23a is the main medicinal ingredient in the traditional Chinese medicinal herb-Rhizoma Alismatis, is safe to organisms, and has good clinical application prospects.
Owner:JIANGNAN UNIV

Catheter-based methods for remodeling a heart chamber

A method for remodeling a heart chamber. A catheter system can be used to deliver implants to the heart chamber. A user can use a handle to adjust the tension of sutures between the implants to adjust the size of the heart chamber. The heart chamber can be remodeled at specific dimensions to address cardiomyopathy in a patient-specific manner.
Owner:INQB8 MEDICAL TECHNOLOGIES LLC

Rapamycin multiphase release or osmotic pump type weekly-effect oral controlled-release composition and preparation method thereof

The invention relates to the technical field of pharmaceutical preparations, in particular to a rapamycin multiphase release or osmotic pump type weekly-effect oral controlled-release composition and a preparation method thereof, and the rapamycin multiphase release or osmotic pump type weekly-effect oral controlled-release composition comprises a quick release layer, a slow release layer and an osmotic pump structural design. According to the application, staged or constant-rate release of rapamycin within 168 hours is realized through a multi-phase release technology or an osmotic pump technology, and the problems that an existing preparation is large in blood concentration fluctuation, high in administration frequency and low in bioavailability are solved. The composition is suitable for treating hypertrophic cardiomyopathy of cats, significantly improves cardiac functions and reduces side effects and risks, is administered once a week, and is convenient to use. The invention further provides a storage method, and the product stability is ensured. The technical scheme has remarkable clinical advantages and application prospects.
Owner:HUZHOU RUNPET BIOTECHNOLOGY CO LTD

Zebra fish model for hypertrophic cardiomyopathy as well as construction method and application of zebra fish model

The invention provides a hypertrophic cardiomyopathy zebrafish model and a construction method and application thereof, and belongs to the technical field of biology, the hypertrophic cardiomyopathy zebrafish model is constructed by overexpressing miR-29a in myocardial cells of zebrafish, and the sequence of the miR-29a is shown as SEQ ID NO.1; the invention further provides a construction method of the hypertrophic cardiomyopathy zebra fish model. The hypertrophic cardiomyopathy zebrafish model Tg (cmlc2: miR-29a, EGFP) with overexpression of miR-29a in myocardial cells is constructed for the first time, and the hypertrophic cardiomyopathy zebrafish model Tg (cmlc2: miR-29a, EGFP) can be used for screening drugs for resisting hypertrophic cardiomyopathy.
Owner:QILU UNIVERSITY OF TECHNOLOGY (SHANDONG ACADEMY OF SCIENCES) +1

Mononuclear cell for treating sepsis cardiomyopathy and application thereof

The invention provides a mononuclear cell for treating sepsis cardiomyopathy, and also relates to application of the mononuclear cell in preparation of drugs for improving sepsis and sepsis cardiomyopathy. The mononuclear cell provided by the invention can have a better targeted enrichment characteristic, so that the effective rate of treatment is improved, and meanwhile, toxic and side effects caused by non-specific distribution of drugs are reduced. Besides, the mononuclear cells are derived from the body of a healthy body, so that the mononuclear cells are more stable in metabolism, higher in absorption efficiency and more excellent in enrichment effect when acting on the body, and the technical problems that in-vivo half-life period of drugs is short and catabolism is easy in the prior art are solved.
Owner:ZHEJIANG UNIV

Processing method and device for hypertrophic cardiomyopathy risk prediction model

PendingCN120565056AMedical data miningHealth-index calculationData setPopulation model
The embodiment of the invention relates to a processing method and device for a hypertrophic cardiomyopathy risk prediction model. The method comprises the following steps: constructing the hypertrophic cardiomyopathy risk prediction model; constructing a generalized crowd model data set as a first data set, and constructing a specified crowd model data set as a second data set based on the specified crowd characteristics; performing pre-training on the model based on the first data set, and then performing fine tuning on the model based on the second data set; after fine tuning is finished, inputting a tested person myocardial strain parameter set meeting the characteristics of the specified crowd into the model for prediction, and feeding back a prediction result to a user. According to the invention, prediction accuracy and prediction stability can be improved.
Owner:SECOND MEDICAL CENT OF CHINESE PLA GENERAL HOSPITAL

Preoperative planning and operation effect evaluation method of HCM, electronic equipment and computer readable medium

The invention discloses a hypertrophic cardiomyopathy (HCM) preoperative planning and operation effect evaluation method based on accurate digital labeling. The method comprises the following steps: performing digital labeling on the thickness of each wall of a left ventricle of a preoperative heart 3D digital model; according to the digital labeling result of the preoperative heart 3D digital model and the normal value of the thickness of the left ventricular wall, a doctor manually depicts the predicted resection range and confirms the predicted retention thickness; measuring the predicted resection volume, the predicted resection area and the left ventricular end diastolic volume after the predicted resection volume is resected; digitally marking the thickness of each wall of the left ventricle of the postoperative heart 3D digital model, and measuring the volume of the corresponding left ventricle in the end diastole; and taking the predicted resection range, the predicted retention thickness, the predicted resection volume, the predicted resection area and the predicted volume as first evaluation data, taking corresponding parameters of the postoperative heart 3D digital model as second evaluation data, and generating an operation evaluation result according to the conformity of the first evaluation data and the second evaluation data.
Owner:THE SIXTH MEDICAL CENT OF THE CHINESE PEOPLES LIBERATION ARMY GENERAL HOSPITAL +1

Bifidobacterium animalis subsp. Lactis capable of relieving liver injury and regulating glycolipid metabolism and application of bifidobacterium animalis subsp. Lactis

The invention relates to the technical field of functional probiotic screening and application, and particularly provides a bifidobacterium animalis subsp. Lactis (Bifidobacterium animalis subsp. Lactis) capable of relieving liver injury and regulating glycolipid metabolism and application of the bifidobacterium animalis subsp. Lactis. The preservation number of the bifidobacterium animalis subsp. Lactis is CGMCC No.31656, and the bifidobacterium animalis subsp. Lactis can effectively reduce blood sugar and blood fat of diabetic mice and relieve insulin resistance and has the capacity of relieving diabetic cardiomyopathy. The strain has wide application prospects in preparation of drugs for treating diabetes mellitus, liver injury and glycolipid metabolism.
Owner:SHENYANG AGRI UNIV

Application of microRNA 244-5P inhibitor in preparation of product for treating sepsis cardiomyopathy

The invention relates to the technical field of medicine preparation, in particular to application of a microRNA244-5P inhibitor in preparation of a product for treating sepsis cardiomyopathy. According to the application of the microRNA 244-5P inhibitor in the preparation of the product for treating the sepsis cardiomyopathy, the sequence of the microRNA 244-5P inhibitor is shown as Seq. ID No. 1, and the microRNA 244-5P inhibitor can be applied to the preparation of the product for treating the sepsis cardiomyopathy. The provided microRNA 244-5P inhibitor lays a precise foundation for targeted treatment of sepsis cardiomyopathy. The inhibitor can specifically recognize and act on related targets, accurately intervene the pathological process of sepsis cardiomyopathy, has more stable structure and activity, can ensure that the inhibition effect can be continuously and efficiently played when a treatment product is prepared, provides a clear material basis and a technical basis for research, development, production and quality control of subsequent products, and has a wide application prospect. The pertinence and the effectiveness of treatment are greatly improved.
Owner:AFFILIATED HOSPITAL OF GUANGDONG MEDICAL UNIV

Disrupting the LINC complex for treating laminopathy

The present invention relates to use of expression vectors and other compounds in methods to disrupt the LInker of Nucleoskeleton and Cytoskeleton (LINC) complex, uncoupling the nucleus from its linkage to the cytoskeleton, resulting in amelioration of diseases caused by one or more Lmna mutations, so-called laminopathies. More particularly, the invention relates to the expression of dominant negative SUN domain protein and / or dominant negative KASH domain protein to disrupt, for example, the LINC complex in cardiomyocytes for suppressing disease progression in dilated cardiomyopathy (DCM).
Owner:AGENCY FOR SCI TECH & RES

Treatment of cardiomyopathy with AAV gene therapy vectors

Provided herein are gene therapy compositions and methods for treating reduced levels of functional cardiac myosin binding protein C in a subject suffering from hypertrophic cardiomyopathy.
Owner:DINAQOR AG

Resmetirom for use in treating cardiomyopathy and / or related heart failure and portal hypertension

The present disclosure provides a method of treating cardiomyopathy and / or related heart failure in a human subject (e.g., an adult human subject) in need of such treatment with 2-(3,5-dichloro-4-((5-isopropyl-6-oxo-1,6-dihydropyridazin-3-yl)oxy)phenyl)-3,5-dioxo-2,3,4,5-tetrahydro-1,2,4-triazine-6-carbonitrile (resmetirom) or a pharmaceutically acceptable salt thereof. The present disclosure also provides a method of treating portal hypertension in a human subject (e.g., an adult human subject) in need of such treatment with 2-(3,5-dichloro-4-((5-isopropyl-6-oxo-1,6-dihydropyridazin-3-yl)oxy)phenyl)-3,5-dioxo-2,3,4,5-tetrahydro-1,2,4-triazine-6-carbonitrile (resmetirom) or a pharmaceutically acceptable salt thereof. The present disclosure also provides a method of treating clinically significant portal hypertension (CSPH) in a human subject (e.g., an adult human subject) in need of such treatment with 2-(3,5-dichloro-4-((5-isopropyl-6-oxo-1,6-dihydropyridazin-3-yl)oxy)phenyl)-3,5-dioxo-2,3,4,5-tetrahydro-1,2,4-triazine-6-carbonitrile (resmetirom) or a pharmaceutically acceptable salt thereof.
Owner:MADRIGAL PHARMACEUTICALS INC

Application of Alismatol B Acetate in the Prevention or Treatment of Hypertrophic Cardiomyopathy

This invention discloses the application of alismazone B acetate in the preparation of drugs for the prevention or treatment of hypertrophic cardiomyopathy and its related symptoms. This invention is the first to propose the use of alismazone B acetate (AB23a) for the prevention or treatment of hypertrophic cardiomyopathy. The invention utilizes AB23a to directly treat cardiomyocytes obtained from directed differentiation of human embryonic stem cells in vitro, finding that AB23a treatment significantly inhibits the hypertrophic phenotype of human embryonic stem cell-cardiomyocytes. Feeding mice with hereditary hypertrophic cardiomyopathy caused by gene mutations to a diet containing AB23a showed that AB23a significantly alleviated pathological myocardial hypertrophy and improved cardiac function in mice. AB23a can be used to prepare drugs against hereditary hypertrophic cardiomyopathy, providing a new approach and method for treating hypertrophic cardiomyopathy. AB23a is the most important medicinal component of the traditional Chinese medicine Alisma plantago-aquatica, is safe for organisms, and has good clinical application prospects.
Owner:JIANGNAN UNIV

Application of nano antibody for blocking RSPO1-LGR4 effect in preparation of medicine for treating cardiovascular diseases

The invention relates to an application of a nano antibody for blocking RSPO1-LGR4 effect in preparation of a medicine for treating cardiovascular diseases, the nano antibody is an NB21 nano antibody, and the NB21 nano antibody has an amino acid sequence as shown in SEQ ID No.12. The invention also relates to an application of the nano antibody for blocking RSPO1-LGR4 effect in preparation of a medicine for treating cardiovascular diseases. The antibody can specifically block a Wnt / beta-catenin pathway, effectively inhibit expression of proinflammatory factors and relieve inflammatory response of heart tissues, so that occurrence and development of cardiovascular diseases are intervened. The nano antibody has significant advantages in the aspects of intervention accuracy, conversion reliability and the like, and preclinical results show that the nano antibody can improve occurrence and development of ischemic cardiomyopathy; the pharmaceutical composition can be used for treating heart failure, myocarditis and adriamycin cardiomyopathy, can be expanded to systemic inflammatory diseases, realizes the trans-boundary application of one drug with multiple targets, fully verifies the anti-inflammatory effectiveness and safety through in-vivo and in-vitro experimental data, provides new targets, diagnostic tools and accurate intervention schemes for diagnosis and treatment of cardiovascular diseases and systemic inflammatory diseases, and has wide application prospects. The obvious scientific innovation significance and clinical transformation value are realized.
Owner:RUIJIN HOSPITAL AFFILIATED TO SHANGHAI JIAO TONG UNIV SCHOOL OF MEDICINE

Application of panthenol-cytochrome c reductase core protein 2 lactic acid modification as therapeutic target in preparation of drugs for treating sepsis cardiomyopathy

The invention relates to the technical field of biological medicines, in particular to application of panthenol-cytochrome c reductase core protein 2 lactic acid modification as a therapeutic target in preparation of drugs for treating sepsis cardiomyopathy. The invention discloses a key effect of Uqcrc2 K109 site lactic acid modification in SIC and application of the Uqcrc2 K109 site lactic acid modification as a therapeutic target. The Uqcrc2-K109 is found to be a therapeutic target of the SIC through molecular biology methods such as proteomics analysis, lactylation omics analysis and co-immunoprecipitation, and the function of a mitochondrial respiratory chain complex III is recovered in a targeted manner by regulating and controlling the lactylation level of the Uqcrc2-K109, so that a new strategy is provided for treatment of the SIC.
Owner:THE SECOND AFFILIATED HOSPITAL OF GUANGZHOU MEDICAL UNIVERSITY

Quinazoline dione compounds and their uses

Quinazoline dione compounds are provided for treating cardiac indications such as hypertrophic cardiomyopathy and diastolic dysfunction. Diseases treated by the methods described herein include, but are not limited to, cardiomyopathy, hypertrophic cardiomyopathy (HCM), abnormal heart rhythms, aortic disease, Marfan syndrome, coronary artery disease, heart attack, heart failure, rheumatic heart disease, peripheral vascular disease, stroke, deep vein thrombosis, and pulmonary embolism.
Owner:EDGEWISE THERAPEUTICS INC

Radiofrequency ablation instrument for treating heart diseases

The invention provides a radiofrequency ablation instrument for treating heart diseases, and relates to the technical field of medical equipment. The radiofrequency ablatometer comprises a man-machine interaction device, a data processing device, a radiofrequency energy generating device, an ablation device, an ablation parameter acquisition device, a twelve-lead ECG signal acquisition device, an ultrasonic image acquisition device, a vital sign monitoring acquisition device and a circulating cooling device which are respectively connected with the radiofrequency energy generating device and the ablation device, the cooling device is used for supplying cooling liquid to the ablation device; the data processing device controls power output of the radio frequency energy generating device according to the twelve-lead ECG signals and ablation parameters, and the man-machine interaction device displays ultrasonic images, vital sign parameters and ECG data. According to the invention, concentrated display and linkage analysis of ultrasonic images, twelve-lead ECG signals and vital sign parameters are realized for the first time, and the device can be widely applied to percutaneous intracardiac radiofrequency ablation treatment of hypertrophic cardiomyopathy, cardiac tumor and other diseases, and has extremely high clinical application value.
Owner:FOURTH MILITARY MEDICAL UNIVERSITY

Application of HIF1A inhibitor in preparation of medicine for preventing and / or treating hypertrophic cardiomyopathy

The invention discloses application of an HIF1A inhibitor in preparation of a medicine for preventing and / or treating hypertrophic cardiomyopathy, and belongs to the field of biological medicine. For a rat model of spontaneous hypertrophic cardiomyopathy caused by MYH7B gene knockout, hypertrophic cardiomyopathy can be significantly relieved by intraperitoneal injection of the HIF1A inhibitor LW6, heart remodeling can be relieved, heart functions can be improved, and myocardial hypertrophy caused by silent MYH7B genes can be effectively resisted by using siRNA of mRNA transcribed by the HIF1A gene. The key mechanism of the HIF1A inhibitor for treating the hypertrophic cardiomyopathy is that the hypertrophic cardiomyopathy is prevented and treated by reducing the expression level of glycolysis key enzymes GLUT1, HK2 and the like, inhibiting the formation and accumulation of cardiac lactic acid and improving the ventricular remodeling of the hypertrophic cardiomyopathy. The results show that the HIF1A inhibitor can be used for treating and / or preventing the human hypertrophic cardiomyopathy and has the potential of being developed into the medicine for clinically preventing and treating the hypertrophic cardiomyopathy.
Owner:TONGJI HOSPITAL ATTACHED TO TONGJI MEDICAL COLLEGE HUAZHONG SCI TECH

Method for treating or preventing hypertrophic cardiomyopathy

One aim of the present invention is to treat or prevent hypertrophic cardiomyopathy. Provided are: a pharmaceutical composition for treatment or prevention of hypertrophic cardiomyopathy comprising a compound inhibiting SGLT1 or a pharmaceutically acceptable salt thereof; and a method of treating or preventing hypertrophic cardiomyopathy, comprising administering a compound inhibiting SGLT1 or a pharmaceutically acceptable salt thereof.
Owner:JAPAN TOBACCO INC

Application of KAT6A inhibitor in preparation of medicine for treating diabetic cardiomyopathy

The invention belongs to the technical field of biological medicines, and particularly discloses application of a KAT6A inhibitor in preparation of a medicine for treating diabetic cardiomyopathy. Specifically, the invention relates to an application of a histone acetyltransferase inhibitor in preparation of drugs for treating or preventing diabetic cardiomyopathy. The invention discloses an application of a KAT6A inhibitor in preparation of a medicine for treating diabetic cardiomyopathy, which is used for treating or preventing diabetic cardiomyopathy by inhibiting acetylation modification of dihydrolipoamide transacetylase protein and inhibiting cell copper death.
Owner:THE FIRST AFFILIATED HOSPITAL OF HAINAN MEDICAL UNIV

Lactobacilli for treating cardiac dysfunction

The use of a bacterium of the species Lactobacillus salivarius in the manufacture of a food product, dietary supplement or medicament for treating cardiac dysfunction, particularly myocardial infarction, congestive heart failure, dilated cardiomyopathy or inflammatory heart disease (including endocarditis, inflammatory cardiomegaly and myocarditis) is disclosed.
Owner:INT N&H DENMARK APS

Neutralization of acyl-coa binding protein for the treatment of cardiac dysfunction

Cardiomyopathies are heart muscle disorders which represent a heterogeneous group of diseases that often lead to progressive heart failure with significant morbidity and mortality. Here, the inventors show that neutralization of acyl CoA binding protein (ACBP) / diazepam binding inhibitor (DBI) reduces the deleterious effect of the Vehicle anthracycline doxombicin on cardiac function. Since anthracycline-induced heart failure and anthracycline-induced senescence are models of accelerated cardiac aging, it is also plausible to use ACBP / DBI inhibition or neutralization as a method to prevent or treat aging of the cardiovascular system. The inventors also found that genetic and pharmacological interventions to deplete ACBP / DBI efficiently improves cardiac dysfunction associated with experimental HFpEF, a condition associated with aging, hypertension and obesity. Accordingly, the present disclosure relates to methods for the treatment of cardiac dysfunction comprising neutralization of ACBP / DBI.
Owner:INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM) +5

4-METHYLSULFONYL-SUBTITUTED PIPERIDINE / UREA COMPOUNDS USEFUL FOR THE TREATMENT OF CARDIAC DISORDERS SUCH AS DYSTIC CARDIOMYOPATHY (DCM)

UndeterminedCY1126200T1Acyl groupHeart disease
The present invention provides novel 4-methylsulfone-substituted piperidine / urea compounds useful for the treatment of dilated cardiomyopathy (DCM) and conditions associated with left and / or right ventricular systolic dysfunction or systolic reserve. The synthesis and characterization of the compounds, as well as methods for the treatment of DCM and other forms of heart disease, are described.
Owner:MYOKARDIA INC

INTERFERING RNA THERAPY FOR PLN-R14del CARDIOMYOPATHY

PendingUS20250346905A1Polymorphism usesDNA/RNA fragmentationPhospholambanDisease
Phospholamban (PLN) is a critical regulator of calcium cyclin and contractility in the heart. The deletion of Arginine 14 of the phospholamban gene (R14del) is associated with the pathogenesis of an inherited form of cardiomyopathy with prominent arrhythmias. Although the genetic etiology is well defined, there are currently no therapies for this rare disease. This disclosure provides an allele-specific silencing approach by interfering RNA (RNAi) to reduce the expression levels of the R14del allele of the PLN gene.
Owner:THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIV +1