Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Peptic Ulcer Disease II: Pathophysiology01:28

Peptic Ulcer Disease II: Pathophysiology

2.0K
Peptic Ulcer Disease (PUD) is characterized by the development of ulcers in the stomach or duodenal mucosa. Its pathophysiology is complex, involving a balance between damaging and protective elements.
Damaging agents such as Helicobacter pylori, gastric acid, pepsin, and nonsteroidal anti-inflammatory drugs (NSAIDs) can weaken the mucosal defense, allowing hydrogen ions to infiltrate back and harm epithelial cells.
2.0K
Coronary Artery Disease II: Pathophysiology01:26

Coronary Artery Disease II: Pathophysiology

380
Coronary Artery Disease (CAD) originates from a series of events that impair the function of coronary arteries, the blood vessels responsible for delivering oxygen-rich blood to the heart muscle. The pathophysiology of CAD is closely linked to atherosclerosis, a chronic inflammatory and lipid-driven condition affecting the vascular endothelium.1. Endothelial DamageThe process begins with damage to the vascular endothelium, which serves as a protective barrier between the blood and the vessel...
380
Gastroesophageal Reflux Disease I: Meaning and Pathophysiology01:29

Gastroesophageal Reflux Disease I: Meaning and Pathophysiology

1.4K
Gastroesophageal Reflux Disease (GERD) involves the recurrent backflow of the stomach or duodenal contents into the esophagus, leading to troublesome symptoms and potential esophageal mucosal damage. Although GERD is often referred to as a disease, it is more accurately described as a syndrome, as it encompasses a range of symptoms and complications rather than a singular pathological entity, impacting a large number of individuals as the most prevalent upper gastrointestinal problem. Roughly...
1.4K
Chronic Obstructive Pulmonary Disease-II: Pathophysiology01:20

Chronic Obstructive Pulmonary Disease-II: Pathophysiology

4.2K
Chronic Obstructive Pulmonary Disease (COPD) pathophysiology is intricate and multifaceted, involving a complex interplay of physiological processes. Understanding these mechanisms is crucial for effectively managing and treating COPD. Here is an in-depth look at the critical elements in the pathophysiology of COPD:
Chronic Inflammation
4.2K
Pathophysiology of Peptic Ulcer Disease: Injurious Factors01:22

Pathophysiology of Peptic Ulcer Disease: Injurious Factors

1.1K
Peptic ulcers are sores on the stomach's inner lining and the upper small intestine, which are the result of disruptions in the mucosal layer that houses parietal cells which produce gastric acid, and chief cells which secrete pepsinogen.
In the antrum region, G cells secrete the gastrin hormone that binds to gastrin-cholecystokinin-B (CCK2) receptors on parietal and enterochromaffin-like (ECL) cells in the fundic glands. Simultaneously, the vagus nerve releases acetylcholine, which binds...
1.1K
Effect of Hepatic Disease on Pharmacokinetics: Pathophysiologic Assessment and Liver Function Test01:22

Effect of Hepatic Disease on Pharmacokinetics: Pathophysiologic Assessment and Liver Function Test

186
In clinical practice, the direct measurement of hepatic blood flow to evaluate liver function presents significant challenges due to the intricate and specialized nature of the necessary techniques. Consequently, healthcare professionals often rely on empirical estimates derived from thorough patient examinations and liver function tests to gauge liver health. Among the tools at their disposal, the Child–Pugh and MELD scoring systems stand out for their ability to categorize and assess...
186

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

H2BE113K mutation promotes breast cancer metastasis through modulating chromatin dynamics.

Science advances·2026
Same author

Treatment of <i>TP53</i>-mutated myelodysplastic syndrome and acute myeloid leukemia with lowintensity metronomic decitabine and venetoclax.

Haematologica·2026
Same author

Pyrimethamine overcomes resistance to hypomethylating agents by reducing de novo pyrimidine synthesis.

Experimental hematology·2026
Same author

Combining drugs that bypass p53 to treat TP53-mutated leukemias.

Blood advances·2025
Same author

Metronomic low-dose regimen of decitabine and venetoclax is safe and reduces monocyte burden in chronic myelomonocytic leukemia.

Haematologica·2025
Same author

Epigenetic drug remedies for SCD root cause pathophysiology.

Blood·2025

Related Experiment Video

Updated: Jan 21, 2026

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
05:23

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

20.3K

Targeting sickle cell disease root-cause pathophysiology with small molecules.

Yogen Saunthararajah1

  • 1Department of Hematology and Oncology, Taussig Cancer Institute, Cleveland Clinic, Cleveland, OH, USA saunthy@ccf.org.

Haematologica
|August 11, 2019
PubMed
Summary

Small molecules offer practical strategies to treat sickle cell disease by preventing sickle hemoglobin polymerization. Current approaches focus on increasing fetal hemoglobin or directly modifying sickle hemoglobin.

More Related Videos

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

10.3K
Dry Root Rot Disease Assays in Chickpea: a Detailed Methodology
06:58

Dry Root Rot Disease Assays in Chickpea: a Detailed Methodology

Published on: January 17, 2021

8.1K

Related Experiment Videos

Last Updated: Jan 21, 2026

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
05:23

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

20.3K
Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

10.3K
Dry Root Rot Disease Assays in Chickpea: a Detailed Methodology
06:58

Dry Root Rot Disease Assays in Chickpea: a Detailed Methodology

Published on: January 17, 2021

8.1K

Area of Science:

  • Hematology
  • Molecular Medicine
  • Drug Discovery

Background:

  • Sickle cell disease stems from sickle hemoglobin polymerization, causing multi-organ damage.
  • Targeting this root cause is key for effective disease modification.

Purpose of the Study:

  • To review small molecule strategies for sickle cell disease treatment.
  • To summarize lessons learned from clinical evaluations of these strategies.

Main Methods:

  • Review of pre-clinical and clinical data on small molecule therapeutics.
  • Analysis of two main therapeutic strategies: fetal hemoglobin induction and direct sickle hemoglobin modification.

Main Results:

  • Two primary small molecule strategies are under clinical evaluation.
  • Fetal hemoglobin induction shifts production away from sickle hemoglobin.
  • Direct chemical modification aims to inhibit polymerization.

Conclusions:

  • Valuable experience has been gained from ongoing research.
  • Future efforts can be guided by current knowledge for practical, accessible treatments.
  • Molecular mechanism-based approaches are crucial for sickle cell disease modification.