Clinical Trials About "About That Base Editing" RSS

02:31 EDT 16th September 2019 | BioPortfolio

We list hundreds of Clinical Trials about "About That Base Editing" on BioPortfolio. We draw our references from global clinical trials data listed on and refresh our database daily.

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Showing "About That Base Editing" Clinical Trials 1–25 of 688


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.

Investigation of the Performance of Ostomy Base Plate

The purpose of this study is to evaluate the performance of SSH (new base plate) on the degree of leakage under the base plate compared to SenSura and Conform 2. Our hypothesis is that SSH will do better than the two comparator products.

Cerebrospinal Acid-base in Pregnant and Non-pregnant Fertile Women

The third trimester of pregnancy is typically characterized by the development of a marked respiratory alkalosis. The possible role of pregnancy-induced variations of cerebrospinal fluid (CSF) acid-base characteristics on the development of hypocapnic alkalosis is still unknown. Aim of this study is to characterize, according to Stewart's approach, the acid-base equilibrium of CSF and arterial plasma of pregnant women and compare the results with data obtained from ferti...

Chairside Time and Bond Failure of Non Custom Versus Custom Base Orthodontic Attachments During Indirect Bonding

compare the chair side time, clinical orthodontic attachment bond failure and accuracy of transfer between non custom attachment base and custom attachment base indirect techniques.

Study to Evaluate the Immunogenicity and Safety of an Ebola Virus (EBOV) Glycoprotein (GP) Vaccine in Healthy Subjects

This is a randomized, observer-blind, placebo-controlled trial in male and female subjects ≥18 to

Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders

Starting from isolating primary cells from affected patients, an in vitro disease model system for KS will be developed. Using alternative strategies to obtain patient-derived mesenchymal stem cells, an integrative approach will be adopted for defining both the transcriptional and epigenetic regulatory networks perturbed upon the loss of function of KMT2D. Combining the self-renewal potential of mesenchymal stem cells (MSCs) with CRISPR/Cas9 technology, an epigenome editing app...

Molecular and Cellular Characterization of Skull Base Tumors

The purpose of the study is to analyze biological samples and genetic material for basic science research investigating the molecular and cellular characterization of skull base tumors.

Loteprednol Etabonate Ophthalmic Base Compared to Loteprednol Etabonate Ophthalmic Suspension vs Placebo in an Allergen Challenge

The purpose of this study is to evaluate the efficacy of loteprednol etabonate ophthalmic base, compared to loteprednol etabonate ophthalmic suspension, and vehicle in the prevention of the signs and symptoms of allergic conjunctivitis in a modified Conjunctival Allergen Challenge model and in an environmental model during pollen season. Comparisons will be made following 2 weeks of dosing.

Feasibility of Stereotactic Image Guidance on the Lateral Skull Base

A clinical trial to asses the accuracy, usefulness and usability of a stereotactic image-guidance system during lateral skull base surgery.

Role of Leukocyte- and Platelet-Rich Fibrin Membranes in Endoscopic Endonasal Skull Base Reconstruction

Prospective investigation of the effectivity of L-PRF membranes for skull base reconstruction after endoscopic endonasal skull base surgery (transsphenoidal) versus classical closure techniques.

Effect of Dried Fruit Intake on Acid-base Balance

Most adults consume acid-producing diets because their high intake of protein and/or cereal grains in relation to their intake of fruits and vegetables. This study is being done to determine whether acid-base balance can be restored by the addition of dried fruits to the diet. In this study adults with low usual fruit intake will be provided with either 100 g per day of a mix of dried fruits or no dried fruit. Participants will be followed for 1 year. Acid-base status will be a...

Non Carbonic Buffer Power of Critical Ill Patients With Sepsis

Alterations of acid-base equilibrium are very common in critically ill patients and understanding their pathophysiology can be important to improve clinical treatment. The human organism is protected against acid-base disorders by several compensatory mechanisms that minimize pH variations in case of blood variations in carbon dioxide content. The aim of the present study is to quantify the buffer power, i.e. the capacity to limit pH variations in response to carbon dioxide ch...

Retention of Metal Reinforced Polyamide Denture Base and Heat Cured Acrylic Resin Denture Base in Complete Mandibular Denture

The trial is done in order to reinforce polyamide to improve its modulus of elasticity to be able to use it as a denture base material in all clinical cases

Impact of Intraoperative Fluid Management on Electrolyte and Acid-base Variables

During major surgical procedures, intravascular volume is maintained with the administration of isotonic fluids such as Lactated Ringers (LR), Normal Saline (NS) or Normosol-R. All three of these fluids are in common clinical use for this purpose. As large volumes of NS may result in a dilutional acidosis and an increasing base deficit, it may cloud clinical decision making when resuscitative efforts are guided by acid-base status on routine arterial blood gas analysis. This is...

Skull Base Reconstruction After Endonasal Cranio-endoscopic Resection Using Autologus Grafts

Endonasal endoscopic approach to the skull base has been expanded in the last several years owing to advances in the radiological aspect that provided a better evaluation of the lesions and the surrounding structures, technological advances that include angled endoscope, development of high-resolution cameras, high definition monitors and navigation systems and better anatomical experience. The endoscopic endonasal approach now provides access to frontal sinus to the second cer...

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...

A Retrospective Comparison of Neonatal Acid-base Status After CD Before January, 2015 and After January, 2016

The objective is to retrospectively gather peri-operative data on neonatal outcomes, primarily neonatal acid-base status, based on umbilical cord gas analysis, for the 23 months preceding and 23 months following the conduct of the "tilt versus supine study". The investigators hypothesize that there will be no difference in mean neonatal umbilical artery base excess in neonates delivered by cesarean section during the period before and after conduct of the study, for elective, u...

Ultrasound Evaluation of OMT for Sacral Base Asymmetry

This prospective, randomized, controlled trial is proposed to investigate the effect of OMT on sacral base asymmetry as assessed by ultrasound.

Efficacy of an Intra-articular Injection of Botulinum Toxin A Associated to Splinting for Base-of-thumb Osteoarthritis

The primary purpose of this study is to determine whether a single ultrasound-guided intra-articular injection of botulinum toxin A associated to splinting is effective in reducing pain at 3 months in base-of-thumb osteoarthritis.

Chlorhexidine Mouthrinses and Plaque Control

The aim of the present study was to compare antiplaque effectiveness of two commercial mouthrinses: 0.12 % Chlorhexidine alcohol base (CLX-A) versus a diluted 0.1% Chlorhexidine non-alcohol base with 5% of Formaldehyde (CLX-F).

Bronchoprovocation Study to Evaluate the Pharmacodynamics of Albuterol Sulfate Inhalation Aerosol, eq 90 mcg Base

This randomized, single-dose, double-blind, double-dummy, four-period, four-sequence, four-treatment, placebo and active controlled, comparative, multiple-center, crossover-design bronchoprovocation study has been designed to evaluate the pharmacodynamic equivalence of albuterol sulfate HFA inhalation aerosol, eq 90 mcg base (Amneal Ireland Limited) to PROAIR® HFA (albuterol sulfate) Inhalation Aerosol, eq 90 mcg base (Teva Respiratory, LLC) in patients with stable mild asthma...

Acid-Base Composition With Use of Hemodialysates

This is a physiology study of two commercially available dialysate acid concentrates. It is a prospective, single center single blind, cross-over, two week investigation of intradialytic acid-base kinetics and physiology using two commercially available acetate hemodialysate compositions in prevalent hemodialysis patients.

Effect of Home-base Exercise With Conical-PEP Device on Physical Performance and Quality of Life in COPD

The aim of this study to investigate the efficacy of a home-base exercise program with a new conical PEP device on physical performance and health related quality of life in COPD patients.

Evaluation of Microbial Growth on Bre-Flex Versus PEEK Denture Base in Bilateral Maxillary Bounded Partial Denture

This study evaluate Microbial growth on Bre-Flex versus PEEK denture base in Bilateral Maxillary bounded partial denture , half of patients will receive a framework with breflex denture base and the other half will receive a framework with PEEK denture base then evaluate the Candida growth

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