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 Experiment Videos

Hemoglobinopathies.

George F Atweh1, Joseph DeSimone, Yogen Saunthararajah

  • 1Mount Sinai Medical Center, New York, NY 10029-6504, USA.

Hematology. American Society of Hematology. Education Program
|November 25, 2003
PubMed
Summary
This summary is machine-generated.

Related Concept Videos

You might also read

Related Articles

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

Sort by
Same author

Bedinvetmab (Librela/Beransa) in dogs raises safety concerns, including rapidly progressive osteoarthritis, and warrants vigilant adverse event reporting.

Journal of the American Veterinary Medical Association·2026
Same author

Associations of testosterone, sex hormone-binding globulin, and related hormones with risks of cancer death, incident cancer, and incident prostate cancer in men: individual participant data meta-analyses.

The lancet. Healthy longevity·2026
Same author

Zero burden multi night monitoring with AI enabled technology reduces obstructive sleep apnea misdiagnosis.

NPJ digital medicine·2026
Same author

New Obstructive Sleep Apnoea Multi-Night Diagnostic Devices. Is a Single Night of Measurement Now Considered Enough?

Respirology (Carlton, Vic.)·2026
Same author

Microneedle array platforms for drug delivery and biomarker sensing: From skin mechanics guided design to scalable manufacture for clinical utility.

Journal of controlled release : official journal of the Controlled Release Society·2026
Same author

Relationships between pre-pandemic mental health, sociodemographic factors and health behaviours in older adults during the acute onset of COVID-19 in Australia: A descriptive analysis.

PloS one·2026
Same journal

Nze C, Flowers CR. Barriers to accessing cellular therapy for patients receiving care in community practices. Hematology Am Soc Hematol Educ Program. 2023;2023(1):382-385.

Hematology. American Society of Hematology. Education Program·2025
Same journal

CAR T-cell therapy and bispecific antibodies in the management of multiple myeloma.

Hematology. American Society of Hematology. Education Program·2025
Same journal

Emerging immunotherapy advances for non-Hodgkin lymphomas: engaging T cells in the fight.

Hematology. American Society of Hematology. Education Program·2025
Same journal

Anticoagulants in hematologic malignancies: what is the data?

Hematology. American Society of Hematology. Education Program·2025
Same journal

Diagnosis and management of cold agglutinin disease.

Hematology. American Society of Hematology. Education Program·2025
Same journal

What to know about rare B-cell malignancies in 2025.

Hematology. American Society of Hematology. Education Program·2025
See all related articles

New therapeutic options for sickle cell disease are emerging, including decitabine and butyrate to increase fetal hemoglobin, and gene therapy in mouse models. Stroke management guidelines for adults are also discussed.

Area of Science:

  • Hematology
  • Genetics
  • Pharmacology

Background:

  • Sickle cell disease (SCD) management has improved, but curative therapies remain limited.
  • Existing treatments like hydroxyurea have limitations in efficacy and accessibility.
  • Novel therapeutic strategies are crucial for improving patient outcomes.

Purpose of the Study:

  • To review emerging therapeutic options for sickle cell disease.
  • To discuss agents that induce fetal hemoglobin (HbF) production.
  • To explore gene therapy and stroke management in SCD.

Main Methods:

  • Review of clinical experiences with decitabine, a hypomethylating agent.
  • Analysis of studies on butyrate as an HbF inducer, alone and in combination with hydroxyurea.

Related Experiment Videos

  • Examination of transgenic sickle mouse models for gene therapy research.
  • Evaluation of stroke prevention and management strategies in adult SCD patients.

    • Main Results:

    • Decitabine shows significant HbF-inducing activity in patients unresponsive to hydroxyurea.
    • Combination therapy with butyrate and hydroxyurea may achieve higher HbF levels.
    • Globin gene transfer in hematopoietic stem cells inhibits sickling in mouse models.
    • Guidelines for adult stroke management in SCD are proposed, distinguishing evidence-based from anecdotal recommendations.

    Conclusions:

    • Emerging therapies like decitabine and butyrate offer promise for increasing HbF levels in SCD.
    • Gene therapy in mouse models demonstrates potential for in vivo correction of sickling.
    • Further research is needed to optimize HbF induction and gene therapy approaches.
    • Evidence-based guidelines are essential for managing stroke in adult SCD patients.