How Gene Therapy is Changing the Future of Medicine

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Күн бұрын

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Chapters
0:00 Introduction
0:31How does gene therapy work?
2:19 What can gene therapy treat?
3:21 Is gene therapy safe?
Gene therapy is a groundbreaking field of medicine that has the potential to revolutionize the way we treat diseases. Gene therapy involves altering or replacing a person's genes to treat or prevent a disease. This approach could offer a cure for genetic disorders that have been previously considered untreatable. Here's everything you need to know about gene therapy and its potential to revolutionize medicine:
How Gene Therapy Works
Gene therapy involves using viruses or other vectors to deliver healthy genes to the body's cells. Once the new genes are introduced, they can help the body produce proteins that it needs to function properly. Gene therapy can be used to correct genetic mutations that cause diseases, such as cystic fibrosis or sickle cell anemia.
Potential Benefits of Gene Therapy
One of the most significant benefits of gene therapy is its potential to treat genetic diseases that have no cure. Gene therapy can provide a permanent cure for these conditions by correcting the underlying genetic defect. Additionally, gene therapy has the potential to treat non-genetic diseases, such as cancer, by using genes that help the body fight off the disease.
Gene Therapy Success Stories
One of the most significant successes in gene therapy is the treatment of spinal muscular atrophy (SMA), a genetic disorder that affects the nervous system and causes muscle weakness. In 2019, the FDA approved a gene therapy called Zolgensma to treat SMA in children under the age of two. The treatment has shown remarkable success, with children showing significant improvements in motor function and survival rates.
Challenges in Gene Therapy
Despite the potential benefits of gene therapy, there are still significant challenges that need to be addressed. One of the biggest challenges is developing safe and effective gene delivery systems that can target specific cells in the body. Additionally, there are concerns about the long-term safety of gene therapy and the potential for unintended consequences, such as triggering an immune response or causing cancer.
Conclusion
Gene therapy has the potential to revolutionize the way we treat diseases, offering hope for those with previously untreatable conditions. While there are still significant challenges to overcome, the successes in gene therapy are a testament to its potential. Gene therapy is a rapidly evolving field of medicine, and we can expect to see many more breakthroughs in the future.
In summary, gene therapy is a promising approach to treating a wide range of diseases and could potentially revolutionize the field of medicine. With continued research and development, we can expect to see significant advancements in the field of gene therapy and the potential for life-changing treatments.

Пікірлер: 30

@Soulkeeper1 7 ай бұрын

As a 41 year old man that has been living with Muscular Dystrophy for the past 30 or so years, I hope gene therapy might finally be something to be hopeful about. I haven't been ambulatory in years and live in a nursing home. This might give me the chance to finally be free.

@MedicalCentric 7 ай бұрын

I understand that living with Muscular Dystrophy for three decades has been challenging, and I empathize with your hope for gene therapy as a potential source of optimism. Gene therapy holds promise in the realm of treating genetic disorders, and ongoing research in the field is continually advancing. While it's essential to maintain realistic expectations, the progress made in gene therapy provides hope for potential breakthroughs in the future. Engaging in discussions with your healthcare team and staying informed about developments in gene therapy may offer insights into any emerging opportunities that could benefit individuals living with Muscular Dystrophy.

@fahimshahriar8378 Жыл бұрын

Medical scientists are urged to do more research on these issues.

@Bigbrokevo Жыл бұрын

I hope they make one for 22q11 deletion syndrome

@seekeroftruth1200 9 ай бұрын

When will this treatment be ready to treat the ATXN2 gene?

@Alex54578 7 ай бұрын

I really hope that gene therapy could cure Hereditary Spastic Paraplegia (spg4)! Being able to walk freely without crutches would be a dream come true!

@MedicalCentric 7 ай бұрын

Gene therapy holds great promise in the field of medical advancements, and there's ongoing research exploring its potential for conditions like Hereditary Spastic Paraplegia (SPG4). As a genetic disorder affecting the nervous system, SPG4 presents challenges, but gene therapy aims to address underlying genetic mutations. If successful, it could offer transformative outcomes, potentially mitigating symptoms and improving mobility for individuals affected by SPG4. The prospect of regaining the ability to walk freely without crutches is indeed a hopeful and life-changing possibility that many are eagerly anticipating as gene therapy continues to progress.

@reneeb724 10 ай бұрын

Could gene therapy be a treatment/cure for FAP genetic disorder? Praying for a cure

@sarahvasig647 2 ай бұрын

excuse me, may I have the permission to use this content for school purposes.🙏

@yesubabuduvvu8007 37 секунд бұрын

Dnm1 gene and nsf gene therapy available ?

@sibu_puhal7255 10 ай бұрын

I am patient spinal muscular atrophy type c 😔🥺please help me

@joy.18 Жыл бұрын

Can you identify the medical applications of gene therapy and stem cells?

@definitlynotbenlente7671 9 ай бұрын

Curing curently untreatable diseases so people can live a normal life

@joshtheflatearthjedi222 9 ай бұрын

Armwrestlers

@user-lz5jc3jh5v Жыл бұрын

Hit like if you are watching this video to prepare for your Gen Bio assignment test!

@umermemunn1171 8 ай бұрын

Is any body guide me about safe sides and how to stop genetic disoder for the next generation

@MedicalCentric 8 ай бұрын

To reduce the risk of genetic disorders in future generations, consider genetic counseling, carrier screening, prenatal testing, and assisted reproductive technologies if needed. Maintaining a healthy lifestyle and staying informed about medical advances can also play a role in genetic health. Consulting with a genetic counselor or healthcare professional is crucial for personalized guidance and informed family planning decisions.

@Kunjufeb.15 8 ай бұрын

Gene therapy done in Alport syndrome?

@MedicalCentric 8 ай бұрын

Gene therapy is an area of ongoing research for Alport syndrome, a genetic condition primarily caused by mutations in specific genes. While there is no widely established gene therapy for Alport syndrome as of my last update in September 2021, researchers are exploring various gene editing techniques to correct the underlying genetic mutations. It's crucial to consult with medical professionals for the most current information on potential gene therapies and their availability.

@m4r0un 7 ай бұрын

Does gene therapy include rare skin disorders than hasn’t responded to any type of treatment?

@MedicalCentric 7 ай бұрын

Yes, gene therapy holds promise for addressing rare skin disorders that have not responded to traditional treatments. In the context of dermatological conditions, gene therapy aims to correct or replace faulty genes responsible for these disorders. By introducing functional genes into the patient's cells, the therapy seeks to restore normal cellular function and alleviate the symptoms of the skin disorder. This approach can be particularly relevant for rare disorders where conventional treatments have proven ineffective. However, it's essential to note that the development and implementation of gene therapies involve rigorous testing and regulatory processes to ensure safety and efficacy before widespread clinical use. While progress has been made, gene therapy for dermatological conditions is an evolving field, and ongoing research is essential to refine and expand its applications.