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Condition: Pain
Therapy: Gene Therapy

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Total 80 results found since Jan 2013.

Fabry disease - current data and therapeutic approaches
Rom J Morphol Embryol. 2021 Jan-Mar;62(1):5-11. doi: 10.47162/RJME.62.1.01.ABSTRACTFabry disease represents an X-linked inherited disorder resulting in the accumulation of globotriaosylceramide (Gb3). This review explains the clinical manifestations and the possible therapies for this condition. Fabry disease is considered the second most frequent lysosomal storage disease. More than 1000 mutations of the galactosidase alpha (GLA) gene associated with this disorder have been identified. Pain, either episodic crises or chronic pain, is one of the earliest symptoms in Fabry disease. Gastrointestinal, ocular, ear or skeletal ...
Source: Romanian Journal of Morphology and Embryology - October 5, 2021 Category: General Medicine Authors: Ilie Robert Dinu Ştefan George Firu Source Type: research

New Directions in Therapeutic Angiogenesis and Arteriogenesis in Peripheral Arterial Disease
Circ Res. 2021 Jun 11;128(12):1944-1957. doi: 10.1161/CIRCRESAHA.121.318266. Epub 2021 Jun 10.ABSTRACTThe prevalence of peripheral arterial disease (PAD) in the United States exceeds 10 million people, and PAD is a significant cause of morbidity and mortality across the globe. PAD is typically caused by atherosclerotic obstructions in the large arteries to the leg(s). The most common clinical consequences of PAD include pain on walking (claudication), impaired functional capacity, pain at rest, and loss of tissue integrity in the distal limbs that may lead to lower extremity amputation. Patients with PAD also have higher t...
Source: Circulation Research - June 10, 2021 Category: Cardiology Authors: Brian H Annex John P Cooke Source Type: research

Sickle cell disease as a vascular disorder.
Authors: Ofori-Acquah SF Abstract Introduction: In sickle cell disease (SCD), hemoglobin S (HbS) red blood cells (RBCs) are characteristically deformed and inflexible. Often breaking down in the circulation, they exhibit increased adhesive properties with the endothelium and activated neutrophils and platelets, increasing the risk of occlusion of the microcirculation. SCD is categorized into two sub-phenotypes: hyperhemolytic, associated with priapism, leg ulcers, pulmonary hypertension, and stroke, and high hemoglobin/viscosity, which may promote vaso-occlusion-associated pain, acute chest syndrome, and osteonecro...
Source: Expert Review of Hematology - May 6, 2020 Category: Hematology Tags: Expert Rev Hematol Source Type: research

Curing hemoglobinopathies: challenges and advances of conventional and new gene therapy approaches.
Inherited hemoglobin disorders, including beta-thalassemia (BT) and sickle-cell disease (SCD) are the most common monogenic diseases worldwide, with a global carrier frequency of over 5%. With migration they are becoming more common worldwide, making their management and care an increasing concern for health care systems.BT is characterized by an imbalance in the α/β-globin chain ratio, ineffective erythropoiesis, chronic hemolytic anemia, and compensatory haemopoietic expansion. Globally, there are over 25,000 births each year with transfusion-dependent thalassemia (TDT). The current available treatment f...
Source: Mediterranean Journal of Hematology and Infectious Diseases - October 30, 2019 Category: Hematology Source Type: research

Connecting Metainflammation and Neuroinflammation Through the PTN-MK-RPTP β/ζ Axis: Relevance in Therapeutic Development
Conclusion The expression of the components of the PTN-MK-RPTPβ/ζ axis in immune cells and in inflammatory diseases suggests important roles for this axis in inflammation. Pleiotrophin has been recently identified as a limiting factor of metainflammation, a chronic pathological state that contributes to neuroinflammation and neurodegeneration. Pleiotrophin also seems to potentiate acute neuroinflammation independently of the inflammatory stimulus while MK seems to play different -even opposite- roles in acute neuroinflammation depending on the stimulus. Which are the functions of MK and PTN in chronic neuroi...
Source: Frontiers in Pharmacology - April 11, 2019 Category: Drugs & Pharmacology Source Type: research

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study
Backgroundβ-globin gene transfer has the potential for substantial clinical benefit in patients with sickle cell disease (SCD). LentiGlobin Drug Product (DP) contains autologous CD34+ hematopoietic stem cells (HSCs) transduced with the BB305 lentiviral vector (LVV), encoding β-globin with an anti-sickling substitution (T87Q). The safety and efficacy of LentiGlobin gene therapy is being evaluated in the ongoing Phase 1 HGB-206 study (NCT02140554). Results in the initial 7 patients treated with LentiGlobin DP from steady state bone marrow harvested (BMH) HSCs using original DP manufacturing process (Group A) demons...
Source: Blood - November 21, 2018 Category: Hematology Authors: Tisdale, J. F., Kanter, J., Mapara, M. Y., Kwiatkowski, J. L., Krishnamurti, L., Schmidt, M., Miller, A. L., Pierciey, F. J., Shi, W., Ribeil, J.-A., Asmal, M., Thompson, A. A., Walters, M. C. Tags: 801. Gene Therapy and Transfer: Gene Therapy for Blood Cell Disorders Source Type: research

830 Fabry rat skin findings demonstrate potential roles of inflammation and lipoatrophy in Fabry disease
Fabry disease is an X-linked lysosomal disease caused by α-galactosidase A deficiency. Patients experience distal extremity pain and often develop renal failure, cardiomyopathy, and stroke. Dermatological manifestations include sweating abnormalities and angiokeratomas. To gain a better understanding of disease mechanisms, we generated a Fabry rat model using CRISPR/Cas9 technology and confirmed the absence of α-galactosidase A activity in tissues. Wild type (WT) and Fabry rats were indistinguishable at young ages (weaning-6 months), but aging (9 months+) Fabry rats developed an unkempt appearance, alopecia, and xeroderma.
Source: Journal of Investigative Dermatology - April 27, 2018 Category: Dermatology Authors: J.J. Miller, O. Sokumbi, P.E. North, N.M. Dahms Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research

755 Mosaic RAS/MAPK variants cause sporadic vascular malformations which respond to targeted therapy
Sporadic vascular malformations (VMs) are complex congenital anomalies of blood vessels which lead to disfigurement, overgrowth, stroke, pain and/or life-threatening bleeding, depending on the vessel size and the body site. Therapeutic options are severely limited and multi-disciplinary management remains challenging. To investigate the pathogenesis of 160 sporadic intracranial and extracranial VMs in which known genetic causes had been excluded, sequencing of affected tissue DNA was undertaken using next generation sequencing optimised for detection of low mutant allele frequency.
Source: Journal of Investigative Dermatology - April 27, 2018 Category: Dermatology Authors: S. Polubothu, L. Al-Olabi, K. Dowsett, K. Andrews, P. Stadnik, R. Knox, W. Baird, M. Glover, C. Moss, A. Thomas, L. Biesecker, R. Semple, E. Patton, V. Kinsler Tags: Genetic Disease, Gene Regulation, and Gene Therapy Source Type: research