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Related Concept Videos

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Line Loss01:10

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The different configurations of source-load connections include wye (star) and delta connections. The relationship between line and phase voltages and currents varies depending on the configuration. When the source is supplying power, it is transmitted through the wires to the load, and during this transmission, some power is absorbed by the wires, leading to line loss.
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Reducing Line Loss01:18

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In a three-phase circuit, line loss is an indicator of energy dissipated as heat due to the resistance of transmission lines. To address this, incorporating transformers into the system—a step-up transformer at the source and a step-down transformer at the load—is a strategic solution. Two three-phase transformers are introduced to improve this.
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Major Losses in Pipes01:28

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Related Experiment Video

Updated: Jan 21, 2026

Neuro-rehabilitation Approach for Sudden Sensorineural Hearing Loss
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Gene Therapy for Human Sensorineural Hearing Loss.

Yin Ren1,2, Lukas D Landegger1,2,3, Konstantina M Stankovic1,2,4,5

  • 1Department of Otolaryngology, Harvard Medical School, Boston, MA, United States.

Frontiers in Cellular Neuroscience
|August 6, 2019
PubMed
Summary

Gene therapy offers new hope for treating genetic hearing loss, but challenges like delivery and targeting must be overcome for clinical success in sensorineural hearing loss (SNHL).

Keywords:
Anc80L65adeno-associated virus (AAV)blood labyrinth barriergene therapynanoparticlesround window nichetumor penetrating peptide

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Area of Science:

  • Otolaryngology
  • Genetics
  • Molecular Biology

Background:

  • Hearing loss is a widespread sensory impairment affecting millions globally, with over half of congenital deafness cases being hereditary.
  • Current pharmacologic treatments for genetic and acquired deafness are limited, necessitating novel therapeutic approaches.
  • Advances in sequencing technologies have identified numerous genetic targets for hearing loss amenable to gene therapy.

Purpose of the Study:

  • To review recent advancements in *in vivo* gene therapy for sensorineural hearing loss (SNHL).
  • To outline the significant challenges hindering the clinical translation of gene therapy for SNHL.

Main Methods:

  • Review of current literature on *in vivo* gene therapy for SNHL.
  • Analysis of progress and challenges in preclinical and translational studies.

Main Results:

  • Significant progress has been made in developing gene therapy vectors for SNHL in animal models.
  • Key hurdles include the blood-labyrinth barrier, delivery vehicle specificity, surgical access, and target validation.

Conclusions:

  • *In vivo* gene therapy holds promise for treating SNHL, but further research is crucial.
  • Overcoming existing challenges is essential for successful clinical application of gene therapy for hearing loss.