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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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Tumor Immunotherapy01:27

Tumor Immunotherapy

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Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
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Cancer Vaccines01:30

Cancer Vaccines

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Cancer treatment vaccines are a rapidly evolving field that offers a promising approach to immunotherapy. Unlike traditional vaccines that prevent diseases, cancer treatment vaccines are designed to treat existing cancers by stimulating the immune system to recognize and attack cancer cells.
Cancer vaccines come in two categories: preventive (prophylactic) and treatment (active). Preventive vaccines, such as the Human Papillomavirus (HPV) vaccine, protect against viruses that cause certain...
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Related Experiment Video

Updated: Nov 17, 2025

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
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Evolving AAV-delivered therapeutics towards ultimate cures.

Xiangjun He1, Brian Anugerah Urip1, Zhenjie Zhang1

  • 1School of Biomedical Sciences, Faculty of Medicine; Institute for Tissue Engineering and Regenerative Medicine (iTERM), The Chinese University of Hong Kong, Shatin N.T., Hong Kong SAR, China.

Journal of Molecular Medicine (Berlin, Germany)
|February 17, 2021
PubMed
Summary

Recombinant adeno-associated virus (AAV) vectors are revolutionizing gene therapy for genetic diseases. Combining AAV with CRISPR gene editing shows promise but requires careful consideration of safety and challenges.

Keywords:
Adeno-associated virusGene editingGene therapyGene transfer

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Engineering and Evolution of Synthetic Adeno-Associated Virus AAV Gene Therapy Vectors via DNA Family Shuffling
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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Research

Background:

  • Recombinant adeno-associated virus (AAV) vectors are highly effective for in vivo gene transfer.
  • Several AAV-based gene therapies have gained regulatory approval for genetic disorders.
  • Advancements in gene editing, particularly CRISPR, are expanding therapeutic possibilities.

Purpose of the Study:

  • To review clinical updates on AAV gene therapies.
  • To discuss the use of AAV for delivering CRISPR gene editing components.
  • To analyze challenges and safety concerns of AAV and CRISPR therapeutics.

Main Methods:

  • Literature review of clinical studies and recent developments.
  • Analysis of AAV vector technology in gene therapy.
  • Examination of CRISPR-Cas gene editing systems delivered via AAV.

Main Results:

  • AAV vectors demonstrate strong efficacy and safety in clinical applications.
  • AAV-CRISPR systems show potential for treating genetic diseases.
  • Clinical applications highlight both achievements and toxicity issues.

Conclusions:

  • AAV-based gene therapy is a rapidly advancing field with approved treatments.
  • AAV-delivered CRISPR offers novel therapeutic strategies for genetic diseases.
  • Ongoing research must address safety concerns and optimize delivery for AAV and CRISPR therapeutics.