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Kabuki syndrome CRIPSR/Cas9 KMT2D

Showing 1 - 25 of >10,000
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Gene Editing as a Therapeutic Approach for Rett Syndrome

Recruiting
  • Rett Syndrome
  • Gene editing in vitro
  • Siena, Italy
    University of Siena
Feb 13, 2023

Exploiting Epigenome Editing in Kabuki Syndrome:New Route

Completed
  • Kabuki Syndrome 1
  • Intervention on primary cultured cells
  • Montpellier, Herault, France
    Arnaud de villeneuve Hospital
Nov 30, 2020

People Who Have Genetic Changes and Features of Autism: Simons

Recruiting
  • 16P11.2 Deletion Syndrome
  • +184 more
    • New York, New York
    • +1 more
    Jan 6, 2023

    Kabuki Syndrome Trial in Baltimore (Modified Atkins diet)

    Recruiting
    • Kabuki Syndrome
    • Modified Atkins diet
    • Baltimore, Maryland
      Kennedy Krieger Institute
    Nov 17, 2022

    Non-hodgkin Lymphoma,B Cell Trial in Beijing (Autologous CD19-STAR-T cell, Fludarabine, Cyclophosphamide)

    Recruiting
    • Non-hodgkin Lymphoma,B Cell
    • Autologous CD19-STAR-T cell
    • +2 more
    • Beijing, Beijing, China
    • +1 more
    Nov 21, 2022

    Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn Trial in Nashville (CTX001)

    Recruiting
    • Beta-Thalassemia
    • +4 more
    • CTX001
    • Nashville, Tennessee
    • +5 more
    Feb 2, 2023

    Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance Trial in Nashville (CTX001)

    Recruiting
    • Sickle Cell Disease
    • +4 more
    • CTX001
    • Philadelphia, Pennsylvania
    • +3 more
    Jan 24, 2023

    Hyperperfusion After Carotid Stenting by Multi-parameter

    Enrolling by invitation
    • Cerebral Artery Stenosis
    • +2 more
    • carotid artery stenting
    • Beijing, Beijing, China
      Department of Vascular Ultrasonography, Xuanwu Hospital of Capit
    Sep 21, 2023

    Carbapenem-Resistant Enterobacteriaceae Infection Trial in Jinan (CAZ/AVI plus Aztreonam, Conventional treatment)

    Recruiting
    • Carbapenem-Resistant Enterobacteriaceae Infection
    • CAZ/AVI plus Aztreonam
    • Conventional treatment
    • Jinan, Shandong, China
      Mingju Hao
    Apr 29, 2023

    Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn Trial in Worldwide (CTX001)

    Active, not recruiting
    • Beta-Thalassemia
    • +4 more
    • CTX001
    • Stanford, California
    • +11 more
    Nov 30, 2022

    Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22);

    Completed
    Has Results
    • Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
    • +10 more
    • Erlotinib Hydrochloride
    • Laboratory Biomarker Analysis
    • Houston, Texas
      M D Anderson Cancer Center
    Dec 18, 2019

    Beta-Thalassemia Trial in China (BRL-101)

    Recruiting
    • Beta-Thalassemia
    • Guangzhou, Guangdong, China
    • +3 more
    Nov 7, 2022

    Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome Trial in France (data collection)

    Recruiting
    • Immune Thrombocytopenia
    • +2 more
    • data collection
    • Amiens, France
    • +8 more
    Jul 7, 2023

    Viral Keratitis, Blindness Eye, Herpes Simplex Virus Infection Trial in Shanghai (BD111 Adult single group Dose)

    Completed
    • Viral Keratitis
    • +3 more
    • BD111 Adult single group Dose
    • Shanghai, Shanghai, China
      Eye & Ent Hospital of Fudan University
    Aug 20, 2022

    Beta-Thalassemia, Thalassemia, Hematologic Diseases Trial (CTX001)

    Not yet recruiting
    • Beta-Thalassemia
    • +6 more
    • CTX001
    • (no location specified)
    Jul 26, 2022

    Transfusion Dependent Beta-Thalassaemia Trial in Guangzhou, Shenzhen, Tianjin (ET-01)

    Active, not recruiting
    • Transfusion Dependent Beta-Thalassaemia
    • ET-01
    • Guangzhou, Guangdong, China
    • +3 more
    May 9, 2022

    Rett Syndrome Trial in Verona (Osteopathic manipulative treatment)

    Completed
    • Rett Syndrome
    • Osteopathic manipulative treatment
    • Verona, Italy
      Centro AIRETT Ricerca e Innovazione (CARI)
    Jan 30, 2023

    CSVD Burden in Carotid Artery Stenosis

    Recruiting
    • Carotid Artery Stenosis
    • Cerebral Small Vessel Diseases
    • carotid artery stenting
    • Hangzhou, Zhejiang, China
      Zhejiang Provincial People's Hospital
    Sep 4, 2023

    CTEPH Trial in Fukuoka (Edoxaban, Warfarin Potassium, Warfarin Potassium )

    Active, not recruiting
    • CTEPH
    • Fukuoka, Japan
      Kyushu University Hospital
    Aug 30, 2022

    Sickle Cell Disease Trial in Los Angeles, Oakland (CRISPR_SCD001)

    Not yet recruiting
    • Sickle Cell Disease
    • Los Angeles, California
    • +1 more
    Apr 25, 2022

    Non Hodgkin's Lymphoma Trial in Beijing (TRAC and Power3 Genes Knock-out Allogeneic CD19-targeting CAR-T cell (ATHENA CAR-T),

    Not yet recruiting
    • Non Hodgkin's Lymphoma
    • TRAC and Power3 Genes Knock-out Allogeneic CD19-targeting CAR-T cell (ATHENA CAR-T)
    • +2 more
    • Beijing, China
    • +2 more
    Aug 23, 2023

    Acute Myelocytic Leukemia, Myelodysplastic Syndrome Trial in Germany (Azacitidine)

    Completed
    • Acute Myelocytic Leukemia
    • Myelodysplastic Syndrome
    • Berlin, Germany
    • +10 more
    Nov 12, 2021

    Stem Cells in NF1 Tumors of the Central Nervous System

    Suspended
    • Neurofibromatosis Type 1
    • Tumors of the Central Nervous System
    • Collection of Stem Cells
    • Washington, District of Columbia
      Children's National Medical Center
    Mar 8, 2022

    Multiparametric Cardiac MRI in Patients Under CAR T-cell Therapy

    Recruiting
    • DLBCL
    • +8 more
    • Cardiac MRI
    • Bonn, NRW, Germany
      University Hospital Bonn
    Jun 11, 2022

    Anxiety, Depression Trial (Metacognitive Therapy)

    Not yet recruiting
    • Anxiety
    • Depression
    • Metacognitive Therapy
    • (no location specified)
    Aug 4, 2023