Clinical Trials About "Gene Editing Revolutionizing Medicine Causing Government Ethics Nightmare" RSS

07:22 EDT 26th September 2018 | BioPortfolio

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Showing "Gene Editing Revolutionizing Medicine Causing Government Ethics Nightmare" Clinical Trials 1–25 of 5,600+


Ascending Dose Study of Genome Editing by the Zinc Finger Protein (ZFP) Therapeutic SB-318 in Subjects With MPS I

The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma iduronidase (IDUA) enzyme activity of ascending doses of SB-318. SB-318 is an intravenously delivered ZFP Therapeutic for genome editing. It inserts a correct copy of the IDUA gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme.

A Randomized Controlled Trial of Doxazosin for Nightmares, Sleep Disturbance, and Non-Nightmare Clinical Symptoms in PTSD

This randomized, double-blind, placebo-controlled trial of doxazosin will assess doxazosin's effectiveness for PTSD nightmares, subjective sleep quality, and non-nightmare PTSD symptoms in adult men and women veterans with full and partial-syndromal PTSD.

Blended-learning in Physiotherapy: Professional Ethics

The aim of this study was to assess the effect of a blended-learning model on physiotherapy students´ attitude, knowledge and opinions towards learning professional ethics. A simple-blind clinical trial was performed. An eight-month blended learning program to teach professional ethics in clinical practices was worked out. An online syllabus and online activities were elaborated, whilst face to face active participation techniques were performed to discuss ethical issues dete...

Proactive Ethics Intervention to Improve Intensive Care Unit (ICU) Care

The primary aim of the study is to demonstrate the value of a preemptive approach to ethics consultation in an ICU. The investigators hope to answer the question: Will proactive ethics interventions by a skilled and experienced ethicist, participating in treatment discussions with physicians and nurses, as well as discussions with patients/surrogates, improve the quality of ICU care experienced by patients requiring prolonged treatment in the ICU (5 days or longer) by increasi...

Safety and Efficacy of Two Once Daily Anti Retroviral Treatment Regimens Along With Anti-tuberculosis Treatment

Protocol Summary Title: Evaluation of safety and efficacy of two different once daily anti-retroviral treatment regimens along with anti-tuberculosis treatment in patients with HIV-1 and tuberculosis - Randomized Controlled Clinical Trial Phase: Phase III trial Population: 180 HIV-1 positive patients with tuberculosis Number of Sites: Four. 1. Tuberculosis Research Centre, Chennai 2. Government Medical College, Vellore 3. Government Hospital...

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

Clinical, Cellular, and Molecular Investigation Into Oculocutaneous Albinism

Oculocutaneous albinism (OCA) is a term used to describe inherited forms of hypopigmentation associated with 1) variable levels of cutaneous hypopigmentation, ocular hypopigmentation, and visual deficits, and 2) involvement of both of the major developmental types of pigmented cells, i.e., melanocytes and retinal pigment epithelium. OCA is considered isolated if it involves only tissues that are normally pigmented. The four known types of isolated oculocutaneous albinism (OCA-1...

TGFB1 And LAMA1 Gene Polymorphisms in High Myopia

The investigators aimed to investigate TGFB1 and LAMA1 gene polymorphisms in children with high myopia in order to determine the genetic basis of large myopic shifts causing severe visual impairment and complications. Seventy-four children with high myopia (≥6 diopters [D]; study group) and 77 emmetropic children (±0.5D; control group) were included. Genetic and polymorphism analyses were performed in the Medical Genetics Laboratory using DNA purified from the patients' blo...

Short Course Intermittent Regimens for the Treatment of HIV-Associated Tuberculosis

Title: Randomized clinical trial to assess the efficacy of short course intermittent regimens for the treatment of HIV-associated tuberculosis Phase: Phase III trial Population: 300 HIV positive patients with tuberculosis. Number of Sites:Four 1. Tuberculosis Research Centre, Chennai 2. Government General Hospital, Chennai 3. Government Hospital of Thoracic Medicine, Tambaram 4. Government Rajaji Hospital, Madurai Study Duration:36 months...

Genomic Study of Dipeptidyl Peptidase-4 (DPP-4) and Glucagon-Like Peptide 1 Receptor (GLP1R) in Chinese and it's Apply in Antidiabetes

With the development of pharmacogenomics and pharmacogenetics, personalized medicine based on genetic are increasingly required clinically. Incretin-based therapy is currently the most popular diabetes treatment. This study mainly research the correlation of incretin-related gene polymorphism and individual differences in the response of these drugs from the following four aspects. 1. . To study Chinese human gene polymorphism's mutation frequency and allele frequency, ...

Exposure, Relaxation, & Rescripting Therapy-Child

Untreated trauma-related nightmares and sleep-related disorders are associated with chronic health problems, burdening both the suffering individual and the health-care system. The study implements an innovative, cost-efficient, nightmare treatment for trauma-exposed children. It is the first randomized clinical trial with children, adapting an efficacious adult therapy to a 5-session nightmare treatment for 5-17 year-olds. Trauma nightmares are a mechanism in development and...

Genomic Sequencing in Determining Treatment in Patients With Metastatic Cancer or Cancer That Cannot Be Removed by Surgery

This pilot clinical trial studies patients' genomic sequencing in determining specific treatments, also called Precision Medicine, in patients with cancer that has spread to other parts of the body (metastatic) and/or cannot be removed by surgery. Examining the genetic code of a patient's tumor, a mutation (a change in the deoxyribonucleic acid [DNA] sequence of a cell or gene) may be identified and matched with available treatment that targets the mutated gene or an alternativ...

Blood Biomarkers in Suicidal Behaviour

Suicidal behavior (SB) is a major public health problem in France, with more than 10,000 suicides and 220,000 suicide attempts per year. According to the commonly accepted model for understanding suicidal behavior, individuals who carry a suicidal act when subjected to stress factors (environmental stress, depression, substance ...) are those which have a specific vulnerability. These vulnerabilities can be considered as clinical parameters (propensity to despair, aggre...

Gene Transfer for Sickle Cell Disease

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood ste...

Study of Brain Response to Emotional Pictures Using a Magnetic Resonance Imaging (fMRI) While on Escitalopram

The purpose of this study is to find out what parts of the brain have increased or decreased activity when people are depressed and how antidepressant medicine changes this activity in depressed patients. The genetic samples collected are to look at variation in a gene (serotonin transporter gene), which affects the functioning of the chemical serotonin in the brain

Procalcitonin (PCT) as a Diagnostic Marker of Bacterial Infection in the Patients Admitted for Fever and/or Inflammatory Syndrome to the Internal Medicine Department

Levels of PCT (a marker of bacterial infection) are highest during sepsis: in fact, PCT is normally produced by the C cells in the thyroid gland. PCT was initially studied by Assicot1 for distinguishing between bacterial meningitis and viral meningitis. The CALC-I gene codes for PCT. In the absence of infection, the extrathyroid mRNA expression of the CALC-I gene is repressed, and expression is restricted to neuroendocrine thyroid and pulmonary cells. Infection induces the ubiq...

Genomic Structural Variation in Cancer Susceptibility

This study will look for new types of gene changes that may be related to cancer in some patients. Some gene changes (mutations) are passed on from parents to offspring (child). Other gene changes are new and are seen for the first time in a child. They are not seen in the parent. Some of these gene changes may cause cancers in the offspring. We will look for gene changes by studying patients with cancer their parents and family members without cancer. In this study, we ...

Phase Ib Trial of MSP3 LSP in Children in Tanzania

This study will evaluate the safety of candidate malaria vaccine MSP3 in children aged 12-24 months in Tanzania in a highland area with low malaria transmission. Written informed consent will be sought from all guardians/parents of potentially participating children. Eligible children will be randomly allocated to receive either the the study vaccine (MSP3 for a total of 30 children)) or the control vaccine (hepatitis B for a total of 15 children). The vaccines will be given i...

Gene Therapy in Treating Patients With Cancer of The Liver

RATIONALE: Inserting the p53 gene into a person's tumor may improve the body's ability to fight liver cancer. PURPOSE: Phase I trial to study the effectiveness of gene therapy with the p53 gene in treating patients who have cancer of the liver that cannot be surgically removed.

Comparative Study of Molecular Markers in Cutaneous Inflammation Between Psoriatic Lesional Skin and Healthy Non-lesional Skin

The project topic consists on re-conciliating the fine tuners of the gene expression "microRNAs" and the immunopathogenic occasions responsible for skin disorders in context of skin infection and inflammation such as psoriasis. The skin is a network of effector cells and molecular mediators that constitute a highly sophisticated "Skin Immune System (SIS) described by Jan D Bos in 1986. The cutaneous homeostasis maintenance is dependent on the cross talk between several immune s...

Phase II Study of Intravenous Rexin-G in Osteosarcoma

Rexin-G is a tumor-targeted gene medicine that is designed to seek out and destroy both primary tumors and metastatic cancers without the side effects of standard chemotherapy. The objectives of the study are: (1) to evaluate the clinical effectiveness of intravenous injections of Rexin-G, a tumor-targeted gene vector, in controlling tumor growth and prolonging life, and (2) to evaluate its over-all safety.

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

OBJECTIVES: I. Develop an approach for treating patients with homozygous familial hypercholesterolemia using gene therapy with autologous hepatocytes transduced with a normal low-density lipoprotein receptor gene.

Aberant Gene Expression Prostate Carcinoma

The goal of the study is to gain a better understanding of the molecular changes responsible for causing prostate cancer and that examination of tissue and blood samples will help in the development of improved screening and therapeutic approaches.

Safety, Blood Levels and Effects of GW642444

GW642444 (the study medicine) is a new experimental medicine for treating asthma and chronic obstructive pulmonary disease (COPD). People with asthma and COPD suffer from breathlessness because the small tubes (bronchioles) that carry air in and out of the lungs become narrow. We hope that the study medicine will work by relaxing the muscles in the airways and help to keep the airways open, and make breathing easier. The study medicine might improve on available treatments b...

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