目前和新兴的药物治疗策略,以应对状细胞贫血
Yogindra Persaud1, Alexis Leonard1, Parul Rai1
1Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
状细胞疾病 (SCD) 管理正在通过新疗法和基因疗法取得进展. 改善疾病管理是至关重要的,因为基因疗法不会对所有患有SCD的个体都可获得.
科学领域:
- 血液学 血液学 血液学
- 遗传学 是一个遗传学.
- 药理学 药理学是指药理学的学科.
背景情况:
- 一个多世纪以来,状细胞病 (SCD) 已经被研究了,但有效的治疗方法仍然有限.
- 尽管进行了广泛的研究,但SCD的最佳医疗管理和治疗选择仍在发展.
研究的目的:
- 审查目前用于SCD的疾病修饰疗法.
- 讨论新兴的治疗策略,特别是SCD的基因疗法.
- 突出持续需要改善疾病管理以及治疗选择的持续需求.
主要方法:
- 关于SCD治疗的当前文献的综述.
- 对疾病修饰治疗的分析,包括胎儿血红蛋白诱导剂,单克隆抗体,抗炎调节剂和酶激活剂.
- 对SCD的基因疗法方法的评估.
主要成果:
- 在过去的十年中,SCD的治疗选择出现了重大进展.
- 一系列疾病修饰疗法正在研究和临床使用中.
- 基因疗法代表了SCD的变革性治疗策略.
结论:
- 虽然基因疗法提供了潜在的治疗方法,但改善疾病管理对于大多数患有SCD的人来说至关重要.
- 未来的治疗策略可能涉及单独或组合使用的新药或重新定位的药物.
- 持续的研究和开发对于提高SCD患者的寿命和生活质量至关重要.
更多相关视频
08:23Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
Published on: November 5, 2019
05:23Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
相关概念视频
Multiple Allele Traits
Treatment for Pulmonary Arterial Hypertension: Receptor Tyrosine Kinase Inhibitors and Calcium Channel Blockers
TKIs, such as imatinib (Gleevec), are particularly effective in tackling the growth and mitogenic factors that become upregulated in PAH patients. These factors contribute to the...
Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
Treatment for Pulmonary Arterial Hypertension: Endothelin Receptor Antagonists
ETs are synthesized through a complex sequence of enzymatic steps, primarily involving an enzyme referred to as endothelin-converting enzyme...
Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
