Animal Research Stories

Browse news and features about how animal models are enabling discoveries at Harvard Medical School and beyond, and meet some of the people who work with animals in our community.

Connie Cepko sits in a chair in her office petting her dog, Stewie
Video: Stephanie Dutchen and Rick Groleau

The Journey toward a Gene Therapy for Retinitis Pigmentosa

Animals can play crucial roles in helping researchers understand disease and develop new treatments. Without mice, Harvard Medical School geneticist Connie Cepko wouldn’t be on the cusp of discovering whether her team has developed the first safe and effective gene therapy for people with a form of genetic blindness called retinitis pigmentosa.

An estimated 1.5 million people worldwide live with retinitis pigmentosa, which starts with loss of night vision and usually progresses to loss of color, peripheral, and/or central vision. There is currently no cure and no way to stop the disease from advancing.

In this video, Cepko shares her team's approach to developing a gene therapy that aims to preserve vision in people with RP and certain other forms of genetic blindness, including some cases of macular degeneration, by counteracting damage to the retina caused by any of hundreds of different genetic mutations.

More news and features

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How Does the Brain Make Decisions?
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Mouse study provides insights into communication between neurons during decision-making
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How the Brain’s Compass Guides the Body
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A study in fruit flies reveals how the brain’s compass and steering regions make course corrections
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HIV Drugs Show Promise in Averting Complications from Bacterial Infections
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Study in mice finds a trigger for devastating condition, hints at possible treatment
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Worm Study Raises Concern About DEET’s Effect on Reproduction
January 4, 2024

Findings suggest need to consider reproductive health alongside prevention of insect-borne diseases
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What Happens in the Brain While Daydreaming?
December 13, 2023

Observations in mice hint at role of daydreams in remodeling the brain
a runner's legs exercising
Some Benefits of Exercise Stem from the Immune System
November 3, 2023

Immune cells mobilized in the muscles during exercise fend off inflammation and boost endurance
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Pursuing Three Gene Therapies for Rare Inherited Disease
October 19, 2023

Researchers hope at least one will treat progressive blindness caused by Usher syndrome type 1F
heart cells labeled with green fluorescent protein
How the Heart Starts Beating
September 27, 2023

Researchers discover that heart cells in developing zebrafish start beating suddenly and all at once
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How Neurons Grow Comfortable in Their Own Skin
August 23, 2023

Sensory neurons develop customized nerve endings based on cues from the skin, study in mice suggests
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Scientists Identify Cells Involved in Correcting Errors During Spatial Navigation
July 19, 2023

Neurons become active when mice exploring a maze correct after making a wrong turn
A blue, pink, and black image of a zebrafish brain
Researchers Uncover Signal Needed for Blood-Brain Barrier
July 12, 2023

The signal, produced by neurons, helps the barrier form and maintain its protective properties
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When It Comes to Immunity, You Are What You Eat
June 28, 2023

Mouse study shows how diet altered by gut microbes spurs development of immune cells
A stylized image of a brain under an alarm clock on a background that is half day and half night
Untangling the Mystery of Sleep
April 11, 2023

Recent sleep research reveals unexpected connections between the brain and gut
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How the Brain Senses Infection
March 8, 2023

Researchers discovered specific airway neurons in mice that alert the brain about the flu
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The Case for Female Mice in Neuroscience Research
March 7, 2023

Findings reveal female mice have more stable exploratory behavior than male mice
Patches of bright pink, purple, and green representing different types of neurons
Pair of Studies Uncover Surprising New Roles for Spinal Cord and Brainstem in Touch
November 23, 2022

New research suggests spinal cord and brainstem are essential for processing touch signals as they travel to the brain
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Study Finds Unexpected Protective Properties of Pain
October 14, 2022

Work in mice illuminates how pain neurons shield the gut from damage
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Building a Better Painkiller
July 15, 2022

On a quest to develop more effective pain medicines, scientists are studying how CBD inhibits pain-sensing neurons
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Scent of a Human
May 3, 2022

Researcher probes mysteries of smell through disease-carrying insects
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Seeing Shapes
December 7, 2021

Understanding brain’s visual system could inform development of better artificial systems
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Intestinal Insight
August 6, 2020

Bile component appears to help lower high blood sugar after bariatric surgery
illustration of a brain
Decoding Touch
August 8, 2019

New research points to potential therapy for abnormal touch sensitivity in autism spectrum disorders