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

The Power Superposition Principle01:19

The Power Superposition Principle

788
Consider a circuit with two sinusoidal voltage sources. Each one influences the circuit independently, and the superposition principle helps us understand the combined effect by adding up the responses from each source.
788
DC Generator01:19

DC Generator

2.7K
An alternator converts mechanical energy into electrical energy that varies sinusoidally, resulting in AC current. Meanwhile, a DC generator converts mechanical energy into electrical energy, which are DC pulses with the same polarity. The construction of a DC generator is similar to that of an alternator, except that the pair of slip rings is replaced by a single split ring, also called a commutator. The commutator functions like a periodic rotary switch; it changes the contacts with the...
2.7K
Force and Momentum01:17

Force and Momentum

19.4K
Force and momentum are intimately related. Force acting over time can change momentum, and Newton's second law of motion can be stated in its most broadly applicable form in terms of momentum. Momentum can be applied to systems where the mass is changing, such as rockets, as well as to systems of constant mass. Also, momentum continues to be a key concept in the study of atomic and subatomic particles in quantum mechanics. One can consider systems with varying mass in some detail; however, the...
19.4K
Resistance01:19

Resistance

6.8K
When a current moves through any conductor, the conductor causes some level of difficulty for the current to flow. The measure of that difficulty is known as the resistance of the material and is represented by R. Every material has its own resistance. In the case of conductors, heat is emitted whenever a current passes through them. Resistance depends on the resistivity of the material. Resistivity is a characteristic of the material used to fabricate electrical components, whereas the...
6.8K
Three Force Member01:27

Three Force Member

1.7K
A rigid body subjected to three forces acting at three points is known as a three-force member. These forces must have concurrent lines of action, except for parallel forces, where the lines of action are parallel.
For example, consider a dumpster connected to a pin support at point A and a pin attached to a hydraulic cylinder at point B.
1.7K
Apparent Weight01:09

Apparent Weight

7.0K
True weight is the measure of the gravitational force acting on an object. However, if the object accelerates, its measured weight is different from its true weight. Similar observations can be made when the object is submerged in water. An object's weight in water is its apparent weight, which is equal to the difference between its true weight and the buoyant forces.
Consider a person standing on a bathroom scale inside an elevator. If the scale is accurate at rest, its reading equals the...
7.0K

You might also read

Related Articles

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

Sort by
Same author

Engineering immune tolerance for AAV gene therapy: The Norse way.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
Same author

Hemophilia A gene therapy: Moving to the next chapter.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
Same author

Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
Same author

Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.

Journal of blood medicine·2026
Same author

Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.

Blood advances·2026
Same author

Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.

Molecular therapy : the journal of the American Society of Gene Therapy·2026

Related Experiment Video

Updated: Apr 29, 2026

Continuous Theta Burst Stimulation of the Posterior Medial Frontal Cortex to Experimentally Reduce Ideological Threat Responses
06:42

Continuous Theta Burst Stimulation of the Posterior Medial Frontal Cortex to Experimentally Reduce Ideological Threat Responses

Published on: September 28, 2018

14.3K

A David promoter with Goliath strength.

David M Markusic1, Roland W Herzog1

  • 1UNIVERSITY OF FLORIDA.

Blood
|May 17, 2014
PubMed
Summary

Researchers developed a novel gene therapy tool, HS-CRM8, for hemophilia treatment. This enhancer significantly boosts factor IX expression in liver cells, offering a promising therapeutic approach.

Area of Science:

  • Gene Therapy
  • Molecular Biology
  • Hematology

Background:

  • Hemophilia treatment often requires lifelong factor replacement therapy.
  • Developing effective gene therapy vectors with high tissue specificity is crucial.
  • Current gene therapy approaches face challenges in achieving sustained therapeutic levels.

Purpose of the Study:

  • To computationally derive and validate a novel hepatocyte-specific cis-regulatory module (HS-CRM8).
  • To assess the efficacy of HS-CRM8 in enhancing gene expression for hemophilia gene therapy.
  • To evaluate the impact of HS-CRM8 on Factor IX (FIX) protein levels and activity in vivo.

Main Methods:

  • Computational derivation of a cis-regulatory module (HS-CRM8).
  • Inclusion of HS-CRM8 upstream of a minimal liver-specific promoter in an adenoassociated virus (AAV) vector.

More Related Videos

Methanol Independent Expression by Pichia Pastoris Employing De-repression Technologies
05:30

Methanol Independent Expression by Pichia Pastoris Employing De-repression Technologies

Published on: January 23, 2019

15.6K
Versatility of Protocols for Resistance Training and Assessment Using Static and Dynamic Ladders in Animal Models
08:31

Versatility of Protocols for Resistance Training and Assessment Using Static and Dynamic Ladders in Animal Models

Published on: December 17, 2021

2.7K

Related Experiment Videos

Last Updated: Apr 29, 2026

Continuous Theta Burst Stimulation of the Posterior Medial Frontal Cortex to Experimentally Reduce Ideological Threat Responses
06:42

Continuous Theta Burst Stimulation of the Posterior Medial Frontal Cortex to Experimentally Reduce Ideological Threat Responses

Published on: September 28, 2018

14.3K
Methanol Independent Expression by Pichia Pastoris Employing De-repression Technologies
05:30

Methanol Independent Expression by Pichia Pastoris Employing De-repression Technologies

Published on: January 23, 2019

15.6K
Versatility of Protocols for Resistance Training and Assessment Using Static and Dynamic Ladders in Animal Models
08:31

Versatility of Protocols for Resistance Training and Assessment Using Static and Dynamic Ladders in Animal Models

Published on: December 17, 2021

2.7K
  • Co-expression of a codon-optimized hyperactive human FIX mutant (FIX Padua) with HS-CRM8.
  • Assessment of systemic FIX protein levels and biological activity.

    • Main Results:

    • HS-CRM8 demonstrated high tissue-specific expression in hepatocytes.
    • Introduction of HS-CRM8 resulted in a >1 log increase in systemic FIX protein levels.
    • Supraphysiological FIX activity was achieved across a range of AAV vector doses.

    Conclusions:

    • HS-CRM8 is an effective enhancer for achieving high tissue-specific expression in liver cells.
    • This novel regulatory element significantly improves FIX protein levels and activity for hemophilia gene therapy.
    • HS-CRM8 represents a promising advancement for AAV-based gene therapy strategies in hemophilia.