Clinical Trials About "Trial Tests CRISPR Gene Editing Treat Sickle Cell" RSS

02:08 EST 23rd February 2020 | BioPortfolio

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Showing "Trial Tests CRISPR Gene Editing Treat Sickle Cell" Clinical Trials 1–25 of 36,000+

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Gene Transfer for Patients With Sickle Cell Disease

The purpose of this study is to determine whether transfer of a fetal hemoglobin gene using a lentivirus vector (gene transfer) into human blood making cells is safe and feasible in patients with sickle cell disease.

Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia

Using sickle cell and thalassemia mouse models, researchers will evaluate the possibility of correcting these disorders by inserting healthy genetic material into the diseased blood cells. Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in the mouse models.

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12 subjects 18 to 35 years of age with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIV-infected Subjects With Hematological Malignances

The investigators performed this study to evaluate the safety and feasibility of transplantation with CRISPR/Cas9 CCR5 gene modified CD34+ hematopoietic stem/progenitor cells for patients that develop AIDS and hematological malignances. Patients will be treated with antiviral therapy (ART) to achieve undetectable HIV-1 virus in peripheral blood before conditioning. CD34+ cells from donors will be infused into the patients after treatment with CRISPR/Cas9 to ablate CCR5 gene.

Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease

This study will determine the best ways to collect, process and store umbilical cord blood from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell disease is an abnormality of the hemoglobin in red blood cells that causes the cells to change shape and clump together, preventing their normal flow in the bloodstream. This impairs blood flow to various organs, and the resulting oxygen deprivation causes organ damage. Cord blood is rich i...

Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease Pilot and Feasibility Trial of Plerixafor for Hematopoietic Stem Cell (HSC) Mobilization in Patients With Sickle Cell Disease

Sickle cell disease (SCD) is one of the most common genetic diseases in the world. In North America, an estimated 2600 babies are born with SCD each year1, and approximately 70,000 to 100,000 individuals of all ages are affected in the United States2. The clinical manifestations of SCD include acute events, such as recurrent debilitating painful crises, as well as life-threatening pulmonary, cardiovascular, renal, and neurologic complications. The only established curative trea...

Stem Cell Gene Therapy for Sickle Cell Disease

This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified bone marrow for adults with severe sickle cell disease.

Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease

Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the gene PKLR and sickle cell disease (SCD) symptoms. That gene helps create a protein that red blood cells need to work normally. Differences in the gene may cause changes in the protein. That can affect how red blood cells function and can add to the effect of SCD. Researchers want to study the differences by looking at DNA....

Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)

'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949. The pathophysiology of SCD is a consequence of abnormal polymerization of sickle hemoglobin (HbS) and its effects on red cell membrane properties, shape, and density, and subsequent critical changes in inflammatory cell and endothelial cell function. Our goal is to understand the impact of CMA abnormalities in SCD, by interrogatin...

Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease

To determine the retinal and choroidal thickness in patients with sickle cell disease compared to age, race matched population without sickle cell disease to allow a better understanding of the clinical manifestations of sickle cell retinopathy. The purpose of this research study is to evaluate the relationship between sickle cell disease and the eye. The research study is recruiting African American population with or without Sickle Cell Disease. The investigator in charge of ...

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease. SCD-101 inhibits sickling of red blood cells containing Hb S (sickle hemoglobin) under low oxygen conditions, the underlying cause of sickle cell disease. SCD-101 may inhibit red blood cell sickling in patients with sickle cell disease and reduce symptoms and slow disease progression. This study will evaluate the safety and antisickling activity o...

Study of Hydroxyurea to Treat Sickle Cell Disease

The aim of this single-center observational study was to evaluate quality of life, clinical effectiveness, and satisfaction in pediatric and young adult patients with sickle cell disease receiving hydroxyurea.

Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy

Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease. Objective: To determine which of two different lab tests i...

Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel

Sickle cell anemia and sickle cell thalassemia are frequent diseases among the israeli arab population. The purpose of this study is to assess the clinical characteristics of the patients in one arab village and the laboratory characteristics in the carriers of this gene based in the screening for pregnant women that is carried out in the population of northern Israel. The results can be useful in order to institute universal screening for sickle cell anemia in northern ...

Cell Therapy for High Risk T-Cell Malignancies Using CD7-Specific CAR Expressed On Autologous T Cells

Patients eligible for this study have a type of blood cancer called T-cell leukemia or lymphoma (lymph gland cancer). The body has different ways of fighting infection and disease. This study combines two different ways of fighting disease with antibodies and T cells. Antibodies are types of proteins that protect the body from bacterial and other diseases. T cells, or T lymphocytes, are special infection-fighting blood cells that can kill other cells including tumor cells. ...

Cerebrovascular Involvement in Sickle Cell Disease - Comprehensive Sickle Cell Center

To continue studies on the two major neurological complications of sickle cell disease (SCD): namely, stroke and chronic encephalopathy.

Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients Wit

The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients with Sickle Cell Disease (SCD)

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...


A Study of IMR-687 in Adult Patients With Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)

A Phase 2a randomized, placebo-controlled, multicenter study of orally administered IMR-687 in adults with Sickle Cell Anaemia (SCA).

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...

Multi-center Study of SC411 for Sickle Cell Disease

The purpose of this study is to determine whether treatment of sickle cell patients with docosahexaenoic omega-3 acid (DHA) is effective in prevention of acute sickle cell crisis.

Clinical Trial to Study the Safety and Tolerability of Memantin Mepha® in Sickle Cell Disease Patients

Symptomatic sickle cell disease is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crisis. Hemolysis, vaso- occlusive and pain crises are hallmarks of this disease and are causative for an important socio-economic burden worldwide, especially in Africa. Aside from allogenic stem cell transplantation, which is rarely available and very expensive, at present there is no curative treatment for patients with sickle cell disease (SCD). Th...

Skin and Blood Research Samples From Healthy Volunteers and Sickle Cell Anemia Patients

The investigators plan to obtain skin and blood samples from healthy volunteers and sickle cell anemia patients to use for research to use homologous recombination to correct β-globin gene mutations in therapeutically useful cells, like autologous induced pluripotent stem cells from sickle cell anemia patients.

Prevention of Cerebral Infarction in Sickle Cell Anemia - Comprehensive Sickle Cell Center

To conduct a prospective study aimed at the early detection and treatment of cerebral vascular disease prior to irreversible brain injury in young children with sickle cell anemia (SCA).

Establishing a Database of People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Data Project [C-Data])

Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes. The purpose of this study is to gather medical information from children and adults with SCD and establish a database so that researchers can contact people to participate in future SCD research studies.

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