Last updated: August 17, 2026

Stem Cells  CRISPR gene editing help identify therapeutic targets for neurological disorders

CRISPR-Cas9 technologies and human pluripotent stem cell technologies  help to identify new therapeutic goals for neurological disorders such as schizophrenia and drug addiction.

 

CRISPR-Cas9 modified stem cells is a universal and high-precision gene editing technology that allows researchers to modify certain parts of the body’s genome by changing sections of the DNA sequence.

Human-associated pluripotent stem cells (iPSCs) are a genetic research tool that can be created from adult human cells, avoiding the need to use tissue from embryos (along with relevant controversies).

Stem cells can be converted into cells of any type in the body, which allows research in several human systems. New applications of these relatively recent technologies facilitate research at the gene-specific level, creating the potential for developing new treatments.

Today’s new results show that:

Adaptation of CRISPR-Cas9 modofied stem cells technology can detect epigenetic “from” and “on” signals for genes that cause cocaine dependence .
The scientists used CRISPR-Cas9 to shed light on why people with the 15q13.3 microdeletion syndrome – a rare human genetic disorder – are more likely to develop brain disorders such as autism spectrum disorder, epilepsy and schizophrenia  .

Using iPSC stem cells, researchers have developed a new model of cellular disease to investigate the neurobiological causes of schizophrenia, which are not well understood.

Other recent findings, which were discussed, show that:

UScientists have established that a specific protein produced by the Zick virus changes the properties of neural stem cells in the developing brain of an infected fetus, potentially causing microcephaly in newborns .
“Today’s results show many of the advances we have made in the use of CRISPR-Cas9 and the human technologies of pluripotent stem cells and the amazing discoveries that have resulted,”.

“Neuroscientists use these new tools for gene editing and molecular tools to develop potential therapeutic goals on several disease fronts.”

 

 

 

Tags: addiction, autism, brain, Cas9, cell, cocaine addiction, CRISPR, DNA, epilepsy, gene, gene editing, genes, genetics, genetic disorder, genome, light, microcephaly, nervous system, neurology, protein, schizophrenia, stem cells , virus, virus of Zika

 

email:  [email protected]

Scientific case review

Scientific Case Review

Would you like to understand whether current clinical programs, recent research developments, or emerging approaches may be relevant to your individual situation?

Share your question with our scientific research team and receive information focused on the areas of current research that may be relevant to your case.

  • Review of the information you provide
  • Relevant research and clinical program information
  • A clear response focused on your situation
What happens next? Send your question, receive a focused scientific review, and get a clear response about research and clinical programs relevant to your situation.
Your scientific review will be prepared by Dr. Helen Melnik, PhD , who has more than 25 years of experience in stem cell research and international clinical programs.

No obligation. Your question will be reviewed confidentially.

Educational and research information only. This service does not constitute medical advice, diagnosis, prescription, or a personalized treatment recommendation.

WhatsApp