More articles about: Gene Therapy
dna strand
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Research sheds new light on gene therapy for blood disorders
Research from experts at Michigan Medicine, the Children’s Hospital of Philadelphia and Penn Medicine is breaking ground on new ways of treating blood disorders, such as sickle cell anemia, through gene therapy.
 smiling child physical therapy signs
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Screening and treatment bring hope for children with spinal muscular atrophy 
One in 50 people carry the gene for the neuromuscular condition.
 stethoscope drawing in blue ink on lined paper with lab note writing on bottom right in yellow and navy
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Advancing Gene Editing With New CRISPR/Cas9 Variant
Researchers report the ability to improve safety and efficacy using a CRISPR-Cas9 variant known as miCas9.
 DNA graphic in white with rainbow colors behind
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10 Studies That Highlight the Importance of Rare Disease Research
Rare Disease Day is celebrated across the globe to raise awareness about rare diseases and how they impact patients’ lives. Michigan Medicine is working to better understand the mechanisms behind these rare diseases.
 Zion sitting on steps outside his home with his parents.
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7-Year-Old Receives New FDA-Approved Retina Gene Therapy
Gene therapy may prevent blindness from rare eye disease Leber congenital amaurosis.
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U-M Team Makes Strides in Tissue Regeneration Research
Groundbreaking new research marrying high-intensity focused ultrasound with genetically modified cells may spur bone and soft tissue regeneration.
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Gene Therapy Treatment Targets Rare Mutation Tied to Blindness
: Advances in gene therapy are yielding new options for treating inherited retinal degenerations, giving retina specialists new tools — and new hope for patients and families.
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3 Ways Genetic Counselors Provide Clarity on Eye Disease
Trained to interpret variations in your DNA, these specialists can help confirm a vision-related diagnosis and guide family members in gauging their own risk.
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New CRISPR-Cas9 Tool Edits Both RNA and DNA Precisely
A Cas9 protein discovered in meningitis bacteria can act as precise "scissors" for both types of genetic material, cutting at a desired spot guided by CRISPR RNAs.