gene therapy tagged posts

High-Tech Method for uniquely Targeted Gene Therapy developed

Selective activation of basolateral amygdala projections to the rostromedial striatum induced using the MNM004 capsid drives fear and anxiety phenotypes. 

Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe that the new technology can be likened to dramatically accelerating evolution from millions of years to weeks.

Several of the new revolutionary treatments that have been used clinically in recent years to treat complex diseases – such as spinal muscular atrophy and enzyme deficiency – are based on gene therapy.

With gene therapy, the genetic material is controlled or altered using biological drugs...

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Gene Therapy durably Reverse Congenital Deafness in mice

The left panel is a schematic representation of the human ear. Sound waves are collected by the outer ear made up of the pinna and ear canal. The middle ear, composed of the eardrum and ossicles, transmits sound waves to the inner ear, which features the cochlea – the hearing organ responsible for transmitting auditory messages to the central nervous system. The right panel shows an immunofluorescence image of the auditory sensory epithelium within an injected cochlea. The inner hair cells have been stained for otoferlin in green. Otoferlin is detected in almost all of these cells. The inset is a high magnification area showing an inner hair cell that has not been transduced.
Credit: © Institut Pasteur

In collaboration with the universities of Miami, Columbia and San Francisco, scient...

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Gene Therapy: T cells target Mutations to fight Solid Tumors:

The T cell receptor on T cells might recognize antigens derived from mutated proteins in cancer cells. Once a foreign "non-self" antigen is detected, the T cell will kill the cancer cell. Credit: Matthias Leisegang.

The T cell receptor on T cells might recognize antigens derived from mutated proteins in cancer cells. Once a foreign “non-self” antigen is detected, the T cell will kill the cancer cell. Credit: Matthias Leisegang.

T cell therapies with less side effects. An international team has successfully modified immune cells to recognize and specifically target tumor cells in mice. Cancer treatments based on the findings would likely have fewer side effects than standard therapies. Although T cells migrate into tumors and recognize antigens, the defense mechanism seems to fail during the formation of tumors. T cells in the tumors are usually inactive and therapeutically almost useless...

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Degenerating Neurons respond to Gene Therapy for Alzheimer’s disease

Alzheimer Disease Pathology and Site of Gene Delivery in a Patient 7 Years After Gene Transfer

Alzheimer Disease Pathology and Site of Gene Delivery in a Patient 7 Years After Gene Transfer Credit: http://archneur.jamanetwork.com/article.aspx?articleid=2427383

The affected neurons displayed heightened growth, axonal sprouting and activation of functional markers after nerve growth factor (NGF) was injected into their brains.The findings are derived from postmortem analyses of 10 patients who participated in phase I clinical trials launched in 2001 to assess whether injected NGF – a protein essential to cellular growth, maintenance and survival – might safely slow or prevent neuronal degeneration in patients with AD.

Cholinergic Neuronal Hypertrophy and Sprouting

Cholinergic Neuronal Hypertrophy and Sprouting Credit: http://archneur.jamanetwork.com/article.aspx?articleid=2427383

Administering NGF directly into the brain – a fir...

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