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Recent popular reports from Yimaike★Reduce tumor recurrence and increase remission rate! Han Weidong team announced the three-year follow-up data of dual-target CAR-T cellsMedClub broke the news ★August·ShanghaiOncolytic virus drug development forum is about to open.
Click on the picture and register now July 20, 2021/MedClub News /--Recently, the Wexner Medical Center of Ohio State University School of Medicine discovered a gene therapy based on AVV2 viral vector that can help patients with rare genetic and neurodegenerative diseases AADC (aromatic L-amino acid decarboxylase) Deficiency children
.
The results of this research were published on Nature Communications
.
Aromatic L-amino acid decarboxylase deficiency (AADCD) is a rare autosomal recessive neurometabolic disease that can cause severe deficiencies of serotonin, dopamine, norepinephrine and epinephrine at the same time
.
The disease mainly appears in early infancy and can cause severe ophthalmological crisis (OGC) and lifelong mobility deficits.
At the same time, it is also related to mood disorders, sleep disorders, and hypotonia
.
According to consensus guidelines published in the Orphanet Journal of Rare Diseases, only about 135 children worldwide have been determined to lack the enzyme that produces dopamine in the central nervous system (CNS)
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Due to the rarity of AADCD, the international clinical expertise for evidence-based management is limited, and related treatment plans vary according to the treatment strategies of different expert centers
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At the same time, as the disease progresses, the response rate of patients to levodopa or dopamine agonists gradually declines.
In this case, researchers are exploring more effective treatment options, including implantation of deep brain stimulation electrodes, cell transplantation, and gene therapy
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In order to restore the ability of the patient's body to produce AADC, the researchers designed an innovative gene therapy that encapsulated the human AADC gene in an AAV2 viral vector, and then injected it into the child's midbrain very slowly.
Area
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Accuracy is important for this therapy, because if healthy brain tissue is exposed to the protein, serious adverse reactions such as epilepsy will occur.
Researchers must accurately monitor the spread and response of the drug through real-time MR (magnetic resonance imaging) technology
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According to reports, all seven subjects tolerated the procedure well without any direct short-term and long-term adverse effects
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▲The MR tracking image results of gene delivery showed that all subjects showed measurable clinical improvement in symptoms and motor function after gene delivery
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OGC has been completely improved in six of the seven subjects
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There are some differences in the speed and degree of improvement in motor function, but among the 5 subjects who were followed up for 18 months after gene delivery, 4 had the ability to sit down independently, and 2 children had the ability to walk
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It is worth noting that some researchers said: Eye crisis is the first symptom that disappears after gene therapy surgery, and it will never recur
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▲Ophthalmic crisis (OGC) and changes in motor function after gene delivery.
AADC insufficiency can cause rare childhood diseases, and the more common disease Parkinson's disease is also related to the loss of AADC activity and reduced dopamine secretion
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This gene therapy provides a framework for other genetic diseases of the human central nervous system.
The iterative improvement of the various components of this method will promote a wider range of applications.
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Ohio State University researchers said that based on the success of early trials in AADC deficiency, they are planning to apply brain delivery gene therapy to neurodegenerative diseases such as Alzheimer's disease and multiple system degeneration (MSA)
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Adeno-associated virus vector (AAV) gene therapy has brought new treatment options for the treatment of common and rare genetic diseases.
In the competition of gene therapy, adeno-associated virus (AAV) vector has become one of the main platforms for gene delivery in vivo.
One
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Because the AAV vector has low immunogenicity and cytotoxicity, can transduce non-dividing cells, and integrates into the host genome at a very low frequency, it is considered by many researchers to be safer
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The recombined and modified AAV vectors have different serotypes, have different host ranges and virus characteristics according to different serotypes, and can effectively act on specific cell types, including dividing and non-dividing cell types
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The user needs to select the appropriate serotype according to the target cell or tissue type
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The most frequently used serotype is AAV2, and other serotypes are gradually being used widely
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Taking central neurodegenerative diseases as an example, due to the existence of the blood-brain barrier (BBB), the treatment of central nervous system diseases is very challenging
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Studies have found that many AAV vectors, especially AAV1, AAV2, AAV5, AAV8 and AAV9, are very useful for neuronal transduction
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Many AAV-mediated treatments have been or are being tested
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Recommended reading: AAV delivers brain-derived neurotrophic factors, and Alzheimer’s gene therapy initiates a phase 1 clinical trialYi Mai Meng broke the news.
In addition, AAV has shown significant therapeutic effects in clinical trials for the treatment of multiple diseases.
Can cause significant adverse immune reactions
.
For example, in terms of hereditary ophthalmological diseases such as achoroid disease caused by REP1 gene mutation, color blindness or total color blindness caused by CNGB3 gene mutation, and hereditary optic neuropathy (LHON) caused by mitochondrial DNA point mutations and ATP generation disorders, the research results show acceptance of AAV The patient's vision improved after gene therapy
.
Recommended reading: Milestones in gene therapy! The first patient with AAV gene therapy LHON successfully administered by Newforth Ophthalmology Department Yimai Meng revealed that AAV is also a viral vector widely used in cancer gene therapy
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A variety of AAV vectors have been used in tumor treatment.
Numerous pre-clinical studies have shown that AAV gene therapy successfully inhibited tumor growth and caused tumor regression
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In addition, AAV is also widely used in the treatment of blood diseases, metabolism and other diseases, showing broad application prospects
.
Reference materials: 1.
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