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Videos de Conceptos Relacionados

Design Example: Frog Muscle Response01:14

Design Example: Frog Muscle Response

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A student is tasked to work on an intriguing experiment involving an RL (Resistor-Inductor) circuit to study the muscle response of a frog's leg to electrical stimulation. The RL circuit plays a crucial role in this experiment, providing the means to control and measure the electrical impulses that trigger muscle contraction.
When the switch connecting the RL circuit is closed, a brief muscle contraction is observed. This is because, at a steady state, the inductor acts like a short...
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Muscle Contraction01:15

Muscle Contraction

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Muscle Coordination and Action01:24

Muscle Coordination and Action

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Muscle coordination is a complex and finely tuned process essential for smooth and purposeful movements like flexion, extension, adduction, abduction, and rotation. The human body orchestrates the actions of various muscles working in concert, each with a specific role. Four functional types describe how muscles work together: agonist, antagonist, synergist, and fixator.
Agonists
Agonist muscles, often called prime movers, are the primary muscles responsible for producing a specific movement....
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Exercise and Muscle Performance01:27

Exercise and Muscle Performance

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Exercise induces a range of adaptations in muscle tissue, depending on the type and duration of activity. Such physical training can be broadly categorized into two types: endurance exercises and resistance exercises.
Endurance exercises
Endurance exercises involve running, swimming, or cycling, which require repetitive movements with low force output. When a person engages in endurance exercise, a few noticeable changes occur in their skeletal muscles. For instance, the number of capillaries...
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Generation of Action Potential in Skeletal Muscles01:24

Generation of Action Potential in Skeletal Muscles

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Every cell in the body maintains a membrane potential due to an uneven distribution of positive and negative charges across its plasma membrane. The membrane potential is measured in millivolts and quantifies the difference in charge across the membrane.
Like neurons, muscle cells are also regarded as excitable due to their capacity to change in response to stimuli, primarily due to voltage-gated ion channels embedded in their plasma membranes, which get activated by alterations in the...
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Muscles that Move the Arm01:31

Muscles that Move the Arm

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Nine muscles are involved in arm movements. Two of these, the pectoralis major and latissimus dorsi, originate from the axial skeleton and are called axial muscles. The other seven originate from the scapula and are called the scapular muscles.
The pectoralis major has two origins. Its clavicular head originates on the medial half of the clavicle. In contrast, the sternocostal head originates on the costal cartilages of ribs 1-6, the sternum, and the aponeurosis of the external oblique of the...
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Author Spotlight: Bridging the Gap Between In Vivo and Ex Vivo Studies with the "Avatar" Technique to Advance Muscle Mechanics Research
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Diseñador AAV músculo hacia arriba

Carsten G Bönnemann1

  • 1Neuromuscular and Neurogenetic Disorders of Childhood Section, Neurogenetics Branch, NINDS, NIH, Bethesda, MD, USA.

Cell
|September 17, 2021
PubMed
Resumen

La evolución dirigida creó una nueva cápside de virus adeno-asociado (AAV). Este AAV9 diseñado apunta al músculo esquelético, mejorando la administración de terapia génica a este órgano principal.

Área de la Ciencia:

  • Biología molecular
  • Terapia génica
  • Virología

Sus antecedentes:

  • Los capsidos del virus adeno-asociado (AAV) están diseñados para mejorar la especificidad de la terapia génica.
  • Apuntar al músculo esquelético, el órgano más grande del cuerpo, es crucial para tratar varios trastornos genéticos.
  • Los serotipos AAV anteriores tienen limitaciones en la eficiencia de la orientación del músculo esquelético.

Objetivo del estudio:

  • Desarrollar un nuevo serotipo AAV para la entrega de genes dirigidos al músculo esquelético.
  • Para utilizar la evolución dirigida para crear una cápside AAV a medida con un tropismo mejorado para el músculo esquelético.

Principales métodos:

  • Se emplearon técnicas de evolución dirigida para modificar las proteínas de la cápside AAV9.
  • Seleccionado para las variantes que contienen un motivo de unión a la integrina RGD- (ácido arginina-glicina-aspártico).

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  • Modificaciones focalizadas dentro de una región hipervariable de la cápside AAV9.

    • Principales resultados:

    • Se generó con éxito una cápside AAV9 modificada con un tropismo mejorado para el músculo esquelético.
    • La cápside diseñada incorpora un heptámero de unión a la integrina que contiene RGD.
    • Esta modificación facilita una mejor orientación del órgano humano más grande.

    Conclusiones:

    • La evolución dirigida es una estrategia eficaz para crear vectores AAV específicos de los tejidos.
    • La nueva variante AAV9 diseñada muestra una promesa para la terapia génica dirigida al músculo esquelético.
    • Este avance podría mejorar significativamente la eficacia de las terapias génicas para las enfermedades relacionadas con los músculos.