Gene therapy for haemophilia

Sharma, Akshay; Mathew, Minu; Sriganesh, Vasumathi; Reiss, Ulrike

doi:10.1002/14651858.cd010822.pub4

Cited by 7 publications

(7 citation statements)

References 52 publications

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“…It is characterised by spontaneous or provoked, often uncontrolled, bleeding that affects joints, muscles and other tissues. 9,10…”

Section: Resultsmentioning

confidence: 99%

“…However, in an extensive Cochrane review published on 27 April 2020, 10 the authors ‘found no trials to provide reliable evidence about the risks or benefits of gene therapy for haemophilia. There is a need for trials that assess the long-term feasibility, success and risks of gene therapy for people with haemophilia’.…”

Section: Resultsmentioning

confidence: 99%

“…It is characterised by spontaneous or provoked, often uncontrolled, bleeding that affects joints, muscles and other tissues. 9,10 Haemophilia is caused by a genetic deficiency of one of two blood coagulation factors: a deficiency of factor VIII in haemophilia A and of factor IX in haemophilia B. The incidence of haemophilia A is around 1 in 5000-10,000 male births and 1 in 60,000 male births for haemophilia B.…”

Section: Gt In the Treatment Of Hereditary Blood Disordersmentioning

confidence: 99%

“…Patients with severe disease have less than 1% coagulant activity of the corresponding factor. 10 The standard treatment for haemophilia is based on the correction of factor VIII and IX deficiency with blood transfusions or blood products. When more than 1% activity of the affected coagulation factor is achieved, the bleeding tendency is largely eliminated.…”

Section: Gt In the Treatment Of Hereditary Blood Disordersmentioning

confidence: 99%

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Gene therapy and editing in the treatment of hereditary blood disorders: Medical and ethical aspects

2021

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Gene therapy and gene editing are revolutionising the treatment of genetic diseases, most notably haematological disorders. This paper evaluates the use of both techniques in hereditary blood disorders. Many studies have been conducted in this field, especially with gene therapy, with very promising results in diseases such as haemophilia, certain haemoglobinopathies and Fanconi anaemia. The application of these techniques in clinical practice and the foreseeable development of these approaches in the coming years suggest that it might be useful to evaluate the results achieved thus far. It is also essential to reflect on the possible bioethical concerns raised by the use of both techniques, especially in terms of safety issues for patients, potential side effects, treatment duration, accessibility and cost of treatment, and the heritability of genetic changes produced if germline cells are used.

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“…It is characterised by spontaneous or provoked, often uncontrolled, bleeding that affects joints, muscles and other tissues. 9,10…”

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Gt In the Treatment Of Hereditary Blood Disordersmentioning

confidence: 99%

Section: Gt In the Treatment Of Hereditary Blood Disordersmentioning

confidence: 99%

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Gene therapy and editing in the treatment of hereditary blood disorders: Medical and ethical aspects

2021

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“…Acetylsalicylic acid should not be used because of its inhibitory effect on platelet aggregation. The use of any nonsteroidal anti-inflammatory drugs (NSAIDs) should be discussed beforehand with the patient's hematologist because of the effect these drugs have on platelet aggregation [40] . There are no restrictions on the type of local anesthetic agent used, although vasoconstrictors may provide additional local hemostasis [3] .…”

Section: Local Anesthesia Considerationsmentioning

confidence: 99%

Untitled

2022

Int. J. Appl. Dent. Sci.

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Introduction: Hemophilia is an is an X-linked recessive inherited disorder. It belongs to the group of hereditary disorders caused by the deficiency of one or more coagulation factors. Objective:To analyze the literature on the considerations in the dental office in the management of the hemophiliac patient, particularly the etiology, clinical characteristics, diagnosis, treatment and dental management. Methodology: In order to carry out this literature review, an electronic search was necessary using PUBMED and Google Scholar with the words "Hemophilia AND dentistry" "etiology, clinical and dental characteristics, diagnosis and treatment" and "coagulation factors". Results: Hemophilia is due to an alteration in one of the genes that determine the way in which the organism produces coagulation factor VIII or IX. It occurs in 1 in 10,000 male births. It manifests as excessive bleeding in the joints, pain and edema, ecchymosis, urinary tract and digestive tract bleeding. It results in prolonged bleeding from wounds, dental extractions and surgery. The diagnosis of hemophilia is made by taking a blood sample and measuring the degree of factor activity. The main treatment for severe hemophilia is to receive specific clotting factor replacement. During the consultation, a detailed medical history should be taken and comprehensive clinical examinations should be ordered appropriately. The patient should provide the following information: type and severity of his hemophilia, medication ingest, whether he requires pre-treatment with factor concentrate, or an antifibrinolytic agent. Conclusion: Hemophilia is an X-linked recessive inherited disorder. It belongs to the group of inherited disorders caused by deficiency of one or more clotting factors. Practice guidelines are needed to improve the diagnostic process, improve the quality of dental care.

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