New Brain Gene Therapies Dramatic Success

In summary, the conversation discusses various successes in gene therapy and the technical advancements that have aided in these successes. One notable advancement is the discovery of more effective viruses that can bypass the blood brain barrier and target specific cells. This was achieved through screening samples from different sources. Additionally, the use of gene therapy in patient treatment has been approved by the FDA, with recent developments showing promising results in regenerating skin for a young boy with a genetic disorder. Other sources, such as the NY Times, also provide further details on this skin replacement therapy.
  • #1
BillTre
Science Advisor
Gold Member
2,486
9,719
This Science magazine news article describes several recent successes and the technical advances that helped them along.

One important advance was finding better viruses that could pass the blood brain barrier and infect more specific sets of cells by screening through a bunch of samples from different places.
Smartly making use of the diversity of nature with a proper search.
 
Last edited:
  • Like
Likes Evo, berkeman, Greg Bernhardt and 1 other person
Biology news on Phys.org
  • #2
It definitely seems like gene therapy is finally beginning to deliver therapies that can be used in patients. Earlier this year, the first gene therapy procedure—modifying a patient's T-cells to help fight cancer—was approved by the FDA (and a second version was just recently approved). This week, research reported in Nature showed that gene therapy could help regenerate the skin of a seven-year-old boy suffering from a genetic disorder that caused him to lose 60% of his skin.
 
Last edited:
  • Like
Likes jim mcnamara, berkeman and BillTre
  • #3
Here (from Science mag news and the NY Times) are a couple more descriptions of the skin replacement therapy.
 
  • Like
Likes Greg Bernhardt

1. What are brain gene therapies and how do they work?

Brain gene therapies involve introducing new genes into the brain to treat genetic disorders or other neurological conditions. These new genes can either replace faulty genes or supplement the brain with proteins that are missing or not being produced in sufficient quantities. This is typically done through a viral vector, which is a modified virus that can deliver the new genes into brain cells.

2. What are some examples of neurological conditions that can be treated with new brain gene therapies?

Some examples of neurological conditions that have shown promising results with new brain gene therapies include Parkinson's disease, Alzheimer's disease, Huntington's disease, and certain types of brain tumors. These therapies may also be used to treat rare genetic disorders such as spinal muscular atrophy and Duchenne muscular dystrophy.

3. What are the potential benefits of new brain gene therapies?

The potential benefits of new brain gene therapies include potentially providing a long-term or permanent treatment for neurological conditions, as well as reducing the need for frequent and invasive procedures. These therapies may also have fewer side effects compared to traditional treatments, and could potentially improve overall quality of life for patients.

4. What are some challenges or risks associated with new brain gene therapies?

Some challenges and risks associated with new brain gene therapies include the potential for off-target effects, where the new genes may unintentionally affect other parts of the brain or body. There is also a risk of immune reactions to the viral vector, and the potential for the new genes to spread beyond the intended target. Additionally, these therapies are still in the early stages of development and may not be accessible or affordable for all individuals who could benefit from them.

5. What is the current status of research and development in the field of new brain gene therapies?

The field of new brain gene therapies is continuously evolving and expanding. There are currently several clinical trials underway to test the safety and effectiveness of these therapies, and many more in the preclinical stage. While some therapies have already been approved for use in certain countries, there is still a need for further research and development to ensure the safety and efficacy of these treatments before they can be widely adopted.

Similar threads

Medical Progress for Gene Therapy and CRISPR against Blood Diseases
    • Biology and Medical
Replies
2
Views
1K
Alzheimer’s May Involve Modified Genes in Brain Cells
    • Biology and Medical
Replies
1
Views
2K
Can current gene editing technologies reverse age related mutations?
    • Biology and Medical
Replies
4
Views
819
COVID COVID-19's Impact on the Brain: New Evidence Emerges
    • Biology and Medical
Replies
2
Views
1K
Medical Gene Therapy Successfully Treats Teenage Boy with Sickle Cell Anemia
    • Biology and Medical
Replies
4
Views
2K
INKT (Invariant natural killer T cells) as a COVID-19 therapy
    • Biology and Medical
Replies
1
Views
787
New type of henipavirus identified - Langya
    • Biology and Medical
Replies
1
Views
1K
Medical Using CRISPR to cut HIV out of infected cells
    • Biology and Medical
Replies
19
Views
8K
New CRISPR-based tool for find-and-replace editing of DNA
    • Biology and Medical
Replies
2
Views
3K
COVID Various approaches to new anti-viral drugs to treat COVID-19
    • Biology and Medical
Replies
14
Views
2K

Hot Threads

Recent Insights

Back
Top