Search clinical trials for: Gene editing

Total 267579 results

Peking Union Medical College Hospital

Not yet recruiting

A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. (GEN6050XIIT)

Duchenne Muscular Dystrophy (DMD)
National Institute of Allergy and Infectious Diseases...

Recruiting

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease

Chronic Granulomatous Disease (CGD) | X-Linked Chronic Granulomatous Disease

United States
Verve Therapeutics, Inc.

Not yet recruiting

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Heterozygous Familial Hypercholesterolemia | Premature Coronary Heart Disease
Verve Therapeutics, Inc.

Not yet recruiting

Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial Hypercholesterolemia

New Zealand, United Kingdom
The 923rd Hospital of Joint Logistics Support Force...
Guangzhou Reforgene Medicine Co., Ltd.

Recruiting

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Hemoglobinopathies | Hereditary Diseases | Alpha Thalassemia Hemoglobin H Constant Spring

China
HuidaGene Therapeutics Co., Ltd.
Eye & ENT Hospital of Fudan University; Tianjin Medical University Eye Hospital

Recruiting

CRISPR/cas13-medIated RNA tarGeting tHerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)

Neovascular Age-related Macular Degeneration(nAMD)

China
HuidaGene Therapeutics Co., Ltd.
Eye & ENT Hospital of Fudan University

Not yet recruiting

a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND) (SOUND)

Congenital Hearing Loss

China
Peking University Third Hospital

Recruiting

Safety and Tolerability Study of Gene Editing Drug ZVS203e in Participants With Retinitis Pigmentosa

Retinitis Pigmentosa

China
University of Siena

Recruiting

Gene Editing as a Therapeutic Approach for Rett Syndrome (MECPer-3D)

Rett Syndrome

Italy
German Cancer Research Center
University Hospital Heidelberg; University Hospital Dresden

Not yet recruiting

Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients With AML (GALAXY33)

Relapsed/Refractory Acute Myeloid Leukemia (AML)

Germany
Beam Therapeutics Inc.

Recruiting

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease (BEACON)

Sickle Cell Disease

United States
Vanderbilt University Medical Center
Johns Hopkins University; Emory University; Children's National Research Institute

Recruiting

Cooperative Assessment of Late Effects for SCD Curative Therapies (COALESCE)

Heart Disease | Sickle Cell Disease | Pulmonary Disease | Renal Disease

United States
Hospital District of Helsinki and Uusimaa
Karolinska Institutet; University of Helsinki; University of Tartu; Folkhälsan... and other collaborators

Recruiting

Epitranscriptomic Blood Biomarkers for Coronary Artery Disease - A Prospective Cohort Study (IHD-EPITRAN) (IHD-EPITRAN)

Coronary Artery Disease | Aortic Valve Stenosis | Ischemic Heart Disease | Acute Myocardial Infarction

Finland
National Institute on Deafness and Other Communication...

Recruiting

Natural History of Autosomal Dominant Hearing Loss

Hearing Loss

United States
Sangamo Therapeutics

Active, not recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (LTFU)

Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II

United States
Verve Therapeutics, Inc.

Active, not recruiting

A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial Hypercholesterolemia

New Zealand, United Kingdom
Sangamo Therapeutics

Terminated

Long-term Follow-up of HIV Subjects Exposed to SB-728-T or SB-728mR-T

HIV-1-infection

United States
Sangamo Therapeutics
Sanofi

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Transfusion Dependent Beta-thalassemia

United States
Homology Medicines, Inc

Terminated

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Phenylketonuria | Phenylketonurias | PAH Deficiency

United States
Sangamo Therapeutics

Terminated

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

MPS I

United States
Sangamo Therapeutics

Terminated

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

Mucopolysaccharidosis II | MPS II

United States
Cure Rare Disease, Inc
University of Massachusetts, Worcester

Active, not recruiting

Treatment of a Single Patient With CRD-TMH-001

Duchenne Muscular Dystrophy

United States
Shanghai BDgene Co., Ltd.
Eye & ENT Hospital of Fudan University

Completed

Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis

Cornea | Herpes Simplex Virus Infection | Viral Keratitis | Blindness Eye

China
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

Hemophilia B

United States
National Human Genome Research Institute (NHGRI)

Completed

Examining the Knowledge, Attitudes, and Beliefs of Sickle Cell Disease Patients, Parents of Patients With Sickle Cell Disease, and Providers Towards the Integration of CRISPR in Clinical Care

Sickle Cell Disease

United States
Seoul St. Mary's Hospital

Unknown

Impact of Video Editing Training for Novice Trainees in Laparoscopic Cholecystectomy

Gallstone; Colic

Korea, Republic of
University Hospital, Montpellier
Association Française contre les Myopathies Telethon

Completed

Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders (Epi-KAB)

Kabuki Syndrome 1

France
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Choroid Plexus Dysfunction in Neurological Diseases (PLEXFOLD)

Metabolic Syndrome | Plexus Choroideus

France
University of Oviedo

Not yet recruiting

Physiotherapy in Patients in a Cardiac Intensive Care Unit

Cardiac Surgery Patients

Spain
Bayer

Not yet recruiting

A Study to Learn About How BAY 3283142 Moves Into, Through, and Out of the Body After a Single Dose in Participants With Reduced Liver Function Compared to Participants With Normal Liver Function

Chronic Kidney Disease | Hepatic Impairment
Seoul National University Hospital

Recruiting

Transversalis Fascia Plane Block for Iliac Bone Graft in Children

Postoperative Pain, Acute

Korea, Republic of
Istanbul Demiroglu Bilim University

Not yet recruiting

Delirium Prevention Guideline for Intensive Care Patients

Delirium | Nurse's Role

Turkey
Qatar University
Hamad Medical Corporation

Not yet recruiting

A Pragmatic Randomized Trial of GDM Diagnosis (UPDATE-GDM)

Pregnancy | Gestational Diabetes | Diagnosis
Tisento Therapeutics

Not yet recruiting

A Phase 2b Study of Zagociguat in Patients With MELAS (PRIZM)

Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)

United States, Australia, Italy, Germany, Canada, United Kingdom
China Medical University Hospital
National Science and Technology Council

Recruiting

Acupressure Therapy on Post-stroke Fatigue, Depression, and Sleep Disturbances in Ischemic Stroke Patients

Ischemic Stroke | Sleep | Circadian Rhythm | Post-stroke Fatigue | Acupressure | Inflammatory Cytokine

Taiwan
University of Auckland, New Zealand
Health Research Council, New Zealand; Auckland City Hospital; Kitea Health Ltd

Not yet recruiting

First-in-human Trial of Home Brain Pressure Measured Using Kitea ICP Sensor, Placed During Hydrocephalus Shunt Surgery. (HomeICP)

Hydrocephalus

New Zealand
HoHo Biotech
Tri-Service General Hospital; Taoyuan Armed Forces General Hospital

Recruiting

To Evaluate the Efficacy of Oral Molecular Hydrogen Supplement in Patients With Chronic Diseases

Chronic Disease | Chronic Inflammation

Taiwan
University of Copenhagen

Recruiting

Home-based E-biking Intervention Among Adolescents With Cerebral Palsy

Cerebral Palsy

Denmark
Kahramanmaras Sutcu Imam University

Not yet recruiting

Reliability, Validity, and Responsiveness of the Turkish Version of Eating Assessment Tool-10 for Patients With Primer Sjögren's Syndrome

Sjogren's Syndrome
Ain Shams University

Not yet recruiting

A Comparative Study Between Ultrasound Guided Erector Spinae Plane Block and Combined Ultrasound Guided Pericapsular Nerve Group and Lateral Femoral Nerve Block Following Total Hip Arthroplasty

Post Operative Pain
Istanbul Demiroglu Bilim University

Not yet recruiting

Assessing the Impact of Monitor Maintenance Package Utilization

Patient Safety | Alarm Fatigue

Turkey
Huashan Hospital

Not yet recruiting

The Effectiveness and Safety of Body Posture in Preventing Postoperative Recurrence for Chronic Subdural Hematoma

Recurrence | Chronic Subdural Hematoma

China
Medical University of Graz

Not yet recruiting

The Effect of Alcohol on Common Tremor Syndromes

Parkinson Disease | Dystonia | Essential Tremor

Austria
Maastricht University Medical Center
Gilead Sciences

Not yet recruiting

Sacituzumab Govitecan and Bevacizumab for NSCLC Brain Metastases

NSCLC Stage IV | Brain Metastases, Adult

Netherlands
Hadeer Gomaa Ebraheem Aly

Recruiting

Prevalence of Muscle and Nerve and Joint Pain

Joint Pain | Muscle Pain | Nerve Pain

Egypt
University of Western Sydney
University of Auckland, New Zealand

Recruiting

Body Surface Gastric Mapping in Patients on Semaglutide

Gastroparesis | Semaglutide-Induced Gastric Motility

Australia
Hoffmann-La Roche

Recruiting

A Study to Evaluate the Safety and Biomarker Effects of RO7269162 in Participants at Risk for or at the Prodromal Stage of Alzheimer's Disease (AD)

Alzheimer's Disease

Korea, Republic of, France
Instituto de Oftalmología Fundación Conde de Valenciana

Recruiting

Comparison of Recurrence Between Mini-SLET and Limbal-Conjunctival Autograft Techniques in Primary Pterygium

Pterygium | Pterygium of Both Eyes | Pterygium of Right Eye | Pterygium of Left Eye

Mexico
Zeenat Qureshi Stroke Institute

Not yet recruiting

Clevidipine for the Antihypertensive Treatment of Acute Intracerebral Hemorrhage (CLUTCH)

Stroke | Hypertension | Intracerebral Hemorrhage
Lakehead University
Illinois Institute of Technology, Chicago, USA

Recruiting

Honest, Open, Proud - College for Post-Secondary Students With Mental Health Challenges

Mental Health Issue | Stigmatization | Disclosure

Canada

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